Gene therapy (English Wikipedia)

Clinical trial number NCT01639690 for "β-Thalassemia Major With Autologous CD34+ Hematopoietic Progenitor Cells Transduced With TNS9.3.55 a Lentiviral Vector Encoding the Normal Human β-Globin Gene" at ClinicalTrials.gov
Clinical trial number NCT02247843 for "Stem Cell Gene Therapy for Sickle Cell Disease" at ClinicalTrials.gov
Clinical trial number NCT00012545 for "Collection and Storage of Umbilical Cord Stem Cells for Treatment of Sickle Cell Disease" at ClinicalTrials.gov
Staff (2 February 2019). "Ascending Dose Study of Genome Editing by the Zinc Finger Nuclease (ZFN) Therapeutic SB-913 in Subjects With MPS II". ClinicalTrials.gov. U.S. National Library of Medicine. Retrieved 7 February 2019.
"Single Ascending Dose Study in Participants With LCA10". clinicaltrials.gov. Retrieved 20 August 2019.

crisprmedicinenews.com (Global: low place; English: low place)

"News: Clinical Trial Update: Positive Data for First Ever In Vivo CRISPR Medicine". CRISPR Medicine. Retrieved 16 December 2021.

dddmag.com (Global: low place; English: low place)

"Gene Therapy for PAD Approved". 6 December 2011. Retrieved 5 August 2015.

doi.org (Global: 2^nd place; English: 2^nd place)

Kaji EH, Leiden JM (February 2001). "Gene and stem cell therapies". JAMA. 285 (5): 545–550. doi:10.1001/jama.285.5.545. PMID 11176856.
Rosenberg SA, Aebersold P, Cornetta K, Kasid A, Morgan RA, Moen R, et al. (August 1990). "Gene transfer into humans--immunotherapy of patients with advanced melanoma, using tumor-infiltrating lymphocytes modified by retroviral gene transduction". The New England Journal of Medicine. 323 (9): 570–578. doi:10.1056/NEJM199008303230904. PMID 2381442.
Gorell E, Nguyen N, Lane A, Siprashvili Z (April 2014). "Gene therapy for skin diseases". Cold Spring Harbor Perspectives in Medicine. 4 (4) a015149. doi:10.1101/cshperspect.a015149. PMC 3968787. PMID 24692191.
Sun M (October 1982). "Martin Cline loses appeal on NIH grant". Science. 218 (4567): 37. Bibcode:1982Sci...218...37S. doi:10.1126/science.7123214. PMID 7123214.
Sheridan C (February 2011). "Gene therapy finds its niche". Nature Biotechnology. 29 (2): 121–128. doi:10.1038/nbt.1769. PMID 21301435. S2CID 5063701.
O'Malley BW, Ledley FD (October 1993). "Somatic gene therapy. Methods for the present and future". Arch Otolaryngol Head Neck Surg. 119 (10): 1100–7. doi:10.1001/archotol.1993.01880220044007. PMID 8398061.
Oldfield EH, Ram Z, Culver KW, Blaese RM, DeVroom HL, Anderson WF (February 1993). "Gene therapy for the treatment of brain tumors using intra-tumoral transduction with the thymidine kinase gene and intravenous ganciclovir". Human Gene Therapy. 4 (1): 39–69. doi:10.1089/hum.1993.4.1-39. PMID 8384892.
Maguire AM, Simonelli F, Pierce EA, Pugh EN, Mingozzi F, Bennicelli J, et al. (May 2008). "Safety and efficacy of gene transfer for Leber's congenital amaurosis". The New England Journal of Medicine. 358 (21): 2240–2248. doi:10.1056/NEJMoa0802315. PMC 2829748. PMID 18441370.
Simonelli F, Maguire AM, Testa F, Pierce EA, Mingozzi F, Bennicelli JL, et al. (March 2010). "Gene therapy for Leber's congenital amaurosis is safe and effective through 1.5 years after vector administration". Molecular Therapy. 18 (3): 643–650. doi:10.1038/mt.2009.277. PMC 2839440. PMID 19953081.
Cideciyan AV, Hauswirth WW, Aleman TS, Kaushal S, Schwartz SB, Boye SL, Windsor EA, Conlon TJ, Sumaroka A, Roman AJ, Byrne BJ, Jacobson SG (August 2009). "Vision 1 year after gene therapy for Leber's congenital amaurosis". The New England Journal of Medicine. 361 (7): 725–727. doi:10.1056/NEJMc0903652. PMC 2847775. PMID 19675341.
Bainbridge JW, Smith AJ, Barker SS, Robbie S, Henderson R, Balaggan K, et al. (May 2008). "Effect of gene therapy on visual function in Leber's congenital amaurosis". The New England Journal of Medicine. 358 (21): 2231–2239. doi:10.1056/NEJMoa0802268. hdl:10261/271174. PMID 18441371.
Fischer A, Hacein-Bey-Abina S, Cavazzana-Calvo M (June 2010). "20 years of gene therapy for SCID". Nature Immunology. 11 (6): 457–460. doi:10.1038/ni0610-457. PMID 20485269. S2CID 11300348.
Ferrua F, Brigida I, Aiuti A (December 2010). "Update on gene therapy for adenosine deaminase-deficient severe combined immunodeficiency". Current Opinion in Allergy and Clinical Immunology. 10 (6): 551–556. doi:10.1097/ACI.0b013e32833fea85. PMID 20966749. S2CID 205435278.
Cartier N, Aubourg P (July 2010). "Hematopoietic stem cell transplantation and hematopoietic stem cell gene therapy in X-linked adrenoleukodystrophy". Brain Pathology. 20 (4): 857–862. doi:10.1111/j.1750-3639.2010.00394.x. PMC 8094635. PMID 20626747. S2CID 24182017.
Ledford H (2011). "Cell therapy fights leukaemia". Nature. doi:10.1038/news.2011.472.
LeWitt PA, Rezai AR, Leehey MA, Ojemann SG, Flaherty AW, Eskandar EN, et al. (April 2011). "AAV2-GAD gene therapy for advanced Parkinson's disease: a double-blind, sham-surgery controlled, randomised trial". The Lancet. Neurology. 10 (4): 309–319. doi:10.1016/S1474-4422(11)70039-4. PMID 21419704. S2CID 37154043.
Pearson S, Jia H, Kandachi K (January 2004). "China approves first gene therapy". Nature Biotechnology. 22 (1): 3–4. doi:10.1038/nbt0104-3. PMC 7097065. PMID 14704685.
Pezzoli D, Chiesa R, De Nardo L, Candiani G (September 2012). "We still have a long way to go to effectively deliver genes!". Journal of Applied Biomaterials & Functional Materials. 10 (2): 82–91. doi:10.5301/JABFM.2012.9707. PMID 23015375. S2CID 6283455.
Gothelf A, Gehl J (November 2012). "What you always needed to know about electroporation based DNA vaccines". Human Vaccines & Immunotherapeutics. 8 (11): 1694–1702. doi:10.4161/hv.22062. PMC 3601144. PMID 23111168.
Urnov FD, Rebar EJ, Holmes MC, Zhang HS, Gregory PD (September 2010). "Genome editing with engineered zinc finger nucleases". Nature Reviews Genetics. 11 (9): 636–646. doi:10.1038/nrg2842. PMID 20717154. S2CID 205484701.
Bak RO, Gomez-Ospina N, Porteus MH (August 2018). "Gene Editing on Center Stage". Trends in Genetics. 34 (8): 600–611. doi:10.1016/j.tig.2018.05.004. PMID 29908711. S2CID 49269023.
Stone D, Niyonzima N, Jerome KR (September 2016). "Genome editing and the next generation of antiviral therapy". Human Genetics. 135 (9): 1071–82. doi:10.1007/s00439-016-1686-2. PMC 5002242. PMID 27272125.
Cross D, Burmester JK (September 2006). "Gene therapy for cancer treatment: past, present and future". Clinical Medicine & Research. 4 (3): 218–27. doi:10.3121/cmr.4.3.218. PMC 1570487. PMID 16988102.
Maeder ML, Gersbach CA (March 2016). "Genome-editing Technologies for Gene and Cell Therapy". Molecular Therapy. 24 (3): 430–46. doi:10.1038/mt.2016.10. PMC 4786923. PMID 26755333.
"First CRISPR therapy dosed". Nature Biotechnology. 38 (4): 382. 1 April 2020. doi:10.1038/s41587-020-0493-4. ISSN 1546-1696. PMID 32265555. S2CID 215406440.
Sherkow JS, Zettler PJ, Greely HT (December 2018). "Is it 'gene therapy'?". Journal of Law and the Biosciences. 5 (3): 786–793. doi:10.1093/jlb/lsy020. PMC 6534757. PMID 31143463.
Asaleh A, Zhou J, Rahmanian N (November 2022). "Letter to the Editor: A Lesson for the Future-How Semantic Ambiguity Led to the Spread of Anti-Scientific Attitudes About COVID-19 mRNA Vaccines". Human Gene Therapy. 33 (21–22): 1213–1216. doi:10.1089/hum.2022.29223.aas (inactive 12 July 2025). PMID 36375123.{{cite journal}}: CS1 maint: DOI inactive as of July 2025 (link)
Williams DA, Orkin SH (April 1986). "Somatic gene therapy. Current status and future prospects". The Journal of Clinical Investigation. 77 (4): 1053–6. doi:10.1172/JCI112403. PMC 424438. PMID 3514670.
Mavilio F, Ferrari G (July 2008). "Genetic modification of somatic stem cells. The progress, problems and prospects of a new therapeutic technology". EMBO Reports. 9 (Suppl 1): S64–69. doi:10.1038/embor.2008.81. PMC 3327547. PMID 18578029.
Mullard A (June 2020). "Gene-editing pipeline takes off". Nature Reviews. Drug Discovery. 19 (6): 367–372. doi:10.1038/d41573-020-00096-y. PMID 32415249. S2CID 218657910.
MacLaren RE, Groppe M, Barnard AR, Cottriall CL, Tolmachova T, Seymour L, Clark KR, During MJ, Cremers FP, Black GC, Lotery AJ, Downes SM, Webster AR, Seabra MC (March 2014). "Retinal gene therapy in patients with choroideremia: initial findings from a phase 1/2 clinical trial". Lancet. 383 (9923): 1129–1137. doi:10.1016/S0140-6736(13)62117-0. PMC 4171740. PMID 24439297.
Dever DP, Bak RO, Reinisch A, Camarena J, Washington G, Nicolas CE, et al. (November 2016). "CRISPR/Cas9 β-globin gene targeting in human haematopoietic stem cells". Nature. 539 (7629): 384–389. Bibcode:2016Natur.539..384D. doi:10.1038/nature20134. PMC 5898607. PMID 27820943.
Gupta RM, Musunuru K (October 2014). "Expanding the genetic editing tool kit: ZFNs, TALENs, and CRISPR-Cas9". The Journal of Clinical Investigation. 124 (10): 4154–61. Bibcode:2014JCliI.124.4154G. doi:10.1172/JCI72992. PMC 4191047. PMID 25271723.
Sanches-da-Silva GN, Medeiros LF, Lima FM (21 August 2019). "The Potential Use of the CRISPR-Cas System for HIV-1 Gene Therapy". International Journal of Genomics. 2019 8458263. doi:10.1155/2019/8458263. PMC 6721108. PMID 31531340.
Bi A, Cui J, Ma YP, Olshevskaya E, Pu M, Dizhoor AM, Pan ZH (April 2006). "Ectopic expression of a microbial-type rhodopsin restores visual responses in mice with photoreceptor degeneration". Neuron. 50 (1): 23–33. doi:10.1016/j.neuron.2006.02.026. PMC 1459045. PMID 16600853.
Song, Xiangrong; Liu, Chao; Wang, Ning; Huang, Hai; He, Siyan; Gong, Changyang; Wei, Yuquan (1 January 2021). "Delivery of CRISPR/Cas systems for cancer gene therapy and immunotherapy". Advanced Drug Delivery Reviews. Delivery of Biomacromolecules for Therapeutic Genome Editing. 168: 158–180. doi:10.1016/j.addr.2020.04.010. ISSN 0169-409X. PMID 32360576. S2CID 218493473.
Nayerossadat N, Maedeh T, Ali PA (2012). "Viral and nonviral delivery systems for gene delivery". Advanced Biomedical Research. 1 (1): 27. doi:10.4103/2277-9175.98152. PMC 3507026. PMID 23210086.
Ginn SL, Amaya AK, Alexander IE, Edelstein M, Abedi MR (May 2018). "Gene therapy clinical trials worldwide to 2017: An update". The Journal of Gene Medicine. 20 (5) e3015. doi:10.1002/jgm.3015. PMID 29575374. S2CID 4966300.
Bulcha JT, Wang Y, Ma H, Tai PW, Gao G (February 2021). "Viral vector platforms within the gene therapy landscape". Signal Transduction and Targeted Therapy. 6 (1) 53. doi:10.1038/s41392-021-00487-6. PMC 7868676. PMID 33558455.
Jensen, Thomas Leth; Gøtzsche, Casper René; Woldbye, David P. D. (2021). "Current and Future Prospects for Gene Therapy for Rare Genetic Diseases Affecting the Brain and Spinal Cord". Frontiers in Molecular Neuroscience. 14 695937. doi:10.3389/fnmol.2021.695937. ISSN 1662-5099. PMC 8527017. PMID 34690692.
Manini A, Abati E, Nuredini A, Corti S, Comi GP (2022). "Adeno-Associated Virus (AAV)-Mediated Gene Therapy for Duchenne Muscular Dystrophy: The Issue of Transgene Persistence". Frontiers in Neurology. 12 814174. doi:10.3389/fneur.2021.814174. PMC 8797140. PMID 35095747.
Sabatino DE, Bushman FD, Chandler RJ, Crystal RG, Davidson BL, Dolmetsch R, et al. (August 2022). "Evaluating the state of the science for adeno-associated virus integration: An integrated perspective". Molecular Therapy. 30 (8): 2646–2663. doi:10.1016/j.ymthe.2022.06.004. PMC 9372310. PMID 35690906.
Campochiaro, P. A.; Avery, R.; Brown, D. M.; Heier, J. S.; Ho, A. C.; Huddleston, S. M.; Jaffe, G. J.; Khanani, A. M.; Pakola, S.; Pieramici, D. J.; Wykoff, C. C.; Van Everen, S. (2024). "Gene therapy for neovascular age-related macular degeneration by subretinal delivery of RGX-314: a phase 1/2a dose-escalation study". Lancet. 403 (10436): 1563–1573. doi:10.1016/S0140-6736(24)00310-6. PMID 38554726.
Khanani, A. M.; Boyer, D. S.; Wykoff, C. C.; Regillo, C. D.; Busbee, B. G.; Pieramici, D.; Danzig, C. J.; Joondeph, B. C.; Major Jr, J. C.; Turpcu, A.; Kiss, S. (2024). "Safety and efficacy of ixoberogene soroparvovec in neovascular age-related macular degeneration in the United States (OPTIC): a prospective, two-year, multicentre phase 1 study". eClinicalMedicine. 67 102394. doi:10.1016/j.eclinm.2023.102394. PMC 10751837. PMID 38152412.
Yin H, Kanasty RL, Eltoukhy AA, Vegas AJ, Dorkin JR, Anderson DG (August 2014). "Non-viral vectors for gene-based therapy". Nature Reviews. Genetics. 15 (8): 541–555. doi:10.1038/nrg3763. PMID 25022906. S2CID 15273455.
Krasilnikova, O.; Yakimova, A.; Ivanov, S.; Atiakshin, D.; Kostin, A.A.; Sosin, D.; Shegay, P.; Kaprin, A.D.; Klabukov, I. (2023). "Gene-Activated Materials in Regenerative Dentistry: Narrative Review of Technology and Study Results". International Journal of Molecular Sciences. 24 (22) 16250. doi:10.3390/ijms242216250. ISSN 1422-0067. PMC 10671237. PMID 38003439.
Foldvari, M.; Chen, D.W.; Nafissi, N.; Calderon, D.; Narsineni, L.; Rafiee, A. (2016). "Non-viral gene therapy: Gains and challenges of non-invasive administration methods". Journal of Controlled Release. 240: 165–190. doi:10.1016/j.jconrel.2015.12.012. ISSN 1873-4995. PMID 26686079.
Bertrand N, Grenier P, Mahmoudi M, Lima EM, Appel EA, Dormont F, et al. (October 2017). "Mechanistic understanding of in vivo protein corona formation on polymeric nanoparticles and impact on pharmacokinetics". Nature Communications. 8 (1) 777. Bibcode:2017NatCo...8..777B. doi:10.1038/s41467-017-00600-w. PMC 5626760. PMID 28974673.
Mennati, Afsaneh; Rostamizadeh, Kobra; Fathi, Mojtaba (1 March 2025). "Dual silencing of integrin αvβ3 receptor and insulin-like growth factor 1 receptor using mPEG-PCL/DDAB hybrid nanoparticle loaded siRNA in breast cancer therapy: An in vitro study on MCF-7 cells". International Journal of Biological Macromolecules. 294 139334. doi:10.1016/j.ijbiomac.2024.139334. ISSN 0141-8130. PMID 39743068.
Goswami R, Subramanian G, Silayeva L, Newkirk I, Doctor D, Chawla K, et al. (24 April 2019). "Gene Therapy Leaves a Vicious Cycle". Frontiers in Oncology. 9 297. doi:10.3389/fonc.2019.00297. PMC 6491712. PMID 31069169.
Wirth T, Parker N, Ylä-Herttuala S (August 2013). "History of gene therapy". Gene. 525 (2): 162–169. doi:10.1016/j.gene.2013.03.137. PMID 23618815.
Singh V, Khan N, Jayandharan GR (May 2022). "Vector engineering, strategies and targets in cancer gene therapy". Cancer Gene Therapy. 29 (5): 402–417. doi:10.1038/s41417-021-00331-7. PMID 33859378. S2CID 233258994.
Anguela XM, High KA (January 2019). "Entering the Modern Era of Gene Therapy". Annual Review of Medicine. 70 (1): 273–288. doi:10.1146/annurev-med-012017-043332. PMID 30477394. S2CID 53750215.
Wang Y, Zhang R, Tang L, Yang L (February 2022). "Nonviral Delivery Systems of mRNA Vaccines for Cancer Gene Therapy". Pharmaceutics. 14 (3): 512. doi:10.3390/pharmaceutics14030512. PMC 8949480. PMID 35335891.
Keam, Susan J. (November 2024). "Afamitresgene Autoleucel: First Approval". Molecular Diagnosis & Therapy. 28 (6): 861–866. doi:10.1007/s40291-024-00749-3. ISSN 1179-2000. PMID 39404764.
Monga I, Qureshi A, Thakur N, Gupta AK, Kumar M (September 2017). "ASPsiRNA: A Resource of ASP-siRNAs Having Therapeutic Potential for Human Genetic Disorders and Algorithm for Prediction of Their Inhibitory Efficacy". G3. 7 (9): 2931–2943. doi:10.1534/g3.117.044024. PMC 5592921. PMID 28696921.
Olowoyeye A, Okwundu CI (November 2020). "Gene therapy for sickle cell disease". The Cochrane Database of Systematic Reviews. 2020 (11) CD007652. doi:10.1002/14651858.CD007652.pub7. PMC 8275984. PMID 33251574.
Scott, Lesley J. (February 2015). "Alipogene tiparvovec: a review of its use in adults with familial lipoprotein lipase deficiency". Drugs. 75 (2): 175–182. doi:10.1007/s40265-014-0339-9. ISSN 1179-1950. PMID 25559420.
Guo, Congting; Ma, Xiaoteng; Gao, Fei; Guo, Yuxuan (9 March 2023). "Off-target effects in CRISPR/Cas9 gene editing". Frontiers in Bioengineering and Biotechnology. 11 1143157. doi:10.3389/fbioe.2023.1143157. ISSN 2296-4185. PMC 10034092. PMID 36970624.
Wu, Shao-Shuai; Li, Qing-Cui; Yin, Chang-Qing; Xue, Wen; Song, Chun-Qing (15 March 2020). "Advances in CRISPR/Cas-based Gene Therapy in Human Genetic Diseases". Theranostics. 10 (10): 4374–4382. doi:10.7150/thno.43360. ISSN 1838-7640. PMC 7150498. PMID 32292501.
Woods NB, Bottero V, Schmidt M, von Kalle C, Verma IM (April 2006). "Gene therapy: therapeutic gene causing lymphoma". Nature. 440 (7088): 1123. Bibcode:2006Natur.440.1123W. doi:10.1038/4401123a. PMID 16641981. S2CID 4372110.
Thrasher AJ, Gaspar HB, Baum C, Modlich U, Schambach A, Candotti F, et al. (September 2006). "Gene therapy: X-SCID transgene leukaemogenicity". Nature. 443 (7109): E5-6, discussion E6-7. Bibcode:2006Natur.443E...5T. doi:10.1038/nature05219. PMID 16988659. S2CID 4301764.
Bak RO, Porteus MH (July 2017). "CRISPR-Mediated Integration of Large Gene Cassettes Using AAV Donor Vectors". Cell Reports. 20 (3): 750–756. doi:10.1016/j.celrep.2017.06.064. PMC 5568673. PMID 28723575.
Raper SE, Chirmule N, Lee FS, Wivel NA, Bagg A, Gao GP, et al. (September 2003). "Fatal systemic inflammatory response syndrome in a ornithine transcarbamylase deficient patient following adenoviral gene transfer". Molecular Genetics and Metabolism. 80 (1–2): 148–58. doi:10.1016/j.ymgme.2003.08.016. PMID 14567964.
Frank KM, Hogarth DK, Miller JL, Mandal S, Mease PJ, Samulski RJ, et al. (July 2009). "Investigation of the cause of death in a gene-therapy trial". The New England Journal of Medicine. 361 (2): 161–9. doi:10.1056/NEJMoa0801066. PMID 19587341.
Kayser B, Mauron A, Miah A (March 2007). "Current anti-doping policy: a critical appraisal". BMC Medical Ethics. 8 2. doi:10.1186/1472-6939-8-2. PMC 1851967. PMID 17394662.
Powell R, Buchanan A (February 2011). "Breaking evolution's chains: the prospect of deliberate genetic modification in humans". The Journal of Medicine and Philosophy. 36 (1): 6–27. doi:10.1093/jmp/jhq057. PMID 21228084.
Baylis F, Robert JS (2004). "The inevitability of genetic enhancement technologies". Bioethics. 18 (1): 1–26. doi:10.1111/j.1467-8519.2004.00376.x. PMID 15168695.
Roco MC, Bainbridge WS (2002). "Converging Technologies for Improving Human Performance: Integrating From the Nanoscale". Journal of Nanoparticle Research. 4 (4): 281–295. Bibcode:2002JNR.....4..281R. doi:10.1023/A:1021152023349. S2CID 136290217.
Allhoff F (March 2005). "Germ-line genetic enhancement and Rawlsian primary goods" (PDF). Kennedy Institute of Ethics Journal. 15 (1): 39–56. CiteSeerX 10.1.1.566.171. doi:10.1353/ken.2005.0007. PMID 15881795. S2CID 14432440. Archived (PDF) from the original on 30 January 2012.
"Ethical issues related to prenatal genetic testing. The Council on Ethical and Judicial Affairs, American Medical Association". Archives of Family Medicine. 3 (7): 633–642. July 1994. doi:10.1001/archfami.3.7.633. PMID 7921302.
Smith KR, Chan S, Harris J (October 2012). "Human germline genetic modification: scientific and bioethical perspectives". Archives of Medical Research. 43 (7): 491–513. doi:10.1016/j.arcmed.2012.09.003. PMID 23072719.
Reardon S (14 February 2017). "US science advisers outline path to genetically modified babies". Nature. doi:10.1038/nature.2017.21474.
Baltimore D, Berg P, Botchan M, Carroll D, Charo RA, Church G, et al. (April 2015). "Biotechnology. A prudent path forward for genomic engineering and germline gene modification". Science. 348 (6230): 36–38. Bibcode:2015Sci...348...36B. doi:10.1126/science.aab1028. PMC 4394183. PMID 25791083.
Lanphier E, Urnov F, Haecker SE, Werner M, Smolenski J (March 2015). "Don't edit the human germ line". Nature. 519 (7544): 410–411. Bibcode:2015Natur.519..410L. doi:10.1038/519410a. PMID 25810189.
Liang P, Xu Y, Zhang X, Ding C, Huang R, Zhang Z, et al. (May 2015). "CRISPR/Cas9-mediated gene editing in human tripronuclear zygotes". Protein & Cell. 6 (5): 363–372. doi:10.1007/s13238-015-0153-5. PMC 4417674. PMID 25894090.
Friedmann T, Roblin R (March 1972). "Gene therapy for human genetic disease?". Science. 175 (4025): 949–955. Bibcode:1972Sci...175..949F. doi:10.1126/science.175.4025.949. PMID 5061866. S2CID 19952096.
Cepko CL, Roberts BE, Mulligan RC (July 1984). "Construction and applications of a highly transmissible murine retrovirus shuttle vector. This Vector is used for entering a cell in the humans cell body". Cell. 37 (3): 1053–1062. doi:10.1016/0092-8674(84)90440-9. PMID 6331674. S2CID 34544709.
Blaese RM, Culver KW, Miller AD, Carter CS, Fleisher T, Clerici M, et al. (October 1995). "T lymphocyte-directed gene therapy for ADA- SCID: initial trial results after 4 years". Science. 270 (5235): 475–480. Bibcode:1995Sci...270..475B. doi:10.1126/science.270.5235.475. PMID 7570001. S2CID 46339645.
Trojan J, Johnson TR, Rudin SD, Ilan J, Tykocinski ML, Ilan J (January 1993). "Treatment and prevention of rat glioblastoma by immunogenic C6 cells expressing antisense insulin-like growth factor I RNA". Science. 259 (5091): 94–97. Bibcode:1993Sci...259...94T. doi:10.1126/science.8418502. PMID 8418502.
Trojan J, Pan YX, Wei MX, Ly A, Shevelev A, Bierwagen M, et al. (2012). "Methodology for Anti-Gene Anti-IGF-I Therapy of Malignant Tumours". Chemotherapy Research and Practice. 2012: 1–12. doi:10.1155/2012/721873. PMC 3287029. PMID 22400112.
Abbott A (April 1992). "Gene therapy. Italians first to use stem cells". Nature. 356 (6369): 465. Bibcode:1992Natur.356..465A. doi:10.1038/356465a0. PMID 1560817. S2CID 4319842.
Cavazzana-Calvo M, Thrasher A, Mavilio F (February 2004). "The future of gene therapy". Nature. 427 (6977): 779–781. Bibcode:2004Natur.427..779C. doi:10.1038/427779a. hdl:11380/307566. PMID 14985734. S2CID 4421364.
Naldini, Luigi; Blömer, Ulrike; Gallay, Philippe; Ory, Daniel; Mulligan, Richard; Gage, Fred H.; Verma, Inder M.; Trono, Didier (12 April 1996). "In Vivo Gene Delivery and Stable Transduction of Nondividing Cells by a Lentiviral Vector". Science. 272 (5259): 263–267. Bibcode:1996Sci...272..263N. doi:10.1126/science.272.5259.263. ISSN 0036-8075. PMID 8602510. S2CID 18997464.
Ott MG, Schmidt M, Schwarzwaelder K, Stein S, Siler U, Koehl U, et al. (April 2006). "Correction of X-linked chronic granulomatous disease by gene therapy, augmented by insertional activation of MDS1-EVI1, PRDM16 or SETBP1". Nature Medicine. 12 (4): 401–409. doi:10.1038/nm1393. PMID 16582916. S2CID 7601162.
Brown BD, Venneri MA, Zingale A, Sergi Sergi L, Naldini L (May 2006). "Endogenous microRNA regulation suppresses transgene expression in hematopoietic lineages and enables stable gene transfer". Nature Medicine. 12 (5): 585–591. doi:10.1038/nm1398. PMID 16633348. S2CID 11114427.
Morgan RA, Dudley ME, Wunderlich JR, Hughes MS, Yang JC, Sherry RM, et al. (October 2006). "Cancer regression in patients after transfer of genetically engineered lymphocytes". Science. 314 (5796): 126–129. Bibcode:2006Sci...314..126M. doi:10.1126/science.1129003. PMC 2267026. PMID 16946036.
Levine BL, Humeau LM, Boyer J, MacGregor RR, Rebello T, Lu X, et al. (November 2006). "Gene transfer in humans using a conditionally replicating lentiviral vector". Proceedings of the National Academy of Sciences of the United States of America. 103 (46): 17372–17377. Bibcode:2006PNAS..10317372L. doi:10.1073/pnas.0608138103. PMC 1635018. PMID 17090675.
Dolgin E (2009). "Colour blindness corrected by gene therapy". Nature. doi:10.1038/news.2009.921.
Cartier N, Hacein-Bey-Abina S, Bartholomae CC, Veres G, Schmidt M, Kutschera I, et al. (November 2009). "Hematopoietic stem cell gene therapy with a lentiviral vector in X-linked adrenoleukodystrophy" (PDF). Science. 326 (5954): 818–823. Bibcode:2009Sci...326..818C. doi:10.1126/science.1171242. PMID 19892975. S2CID 27783.
Komáromy AM, Alexander JJ, Rowlan JS, Garcia MM, Chiodo VA, Kaya A, et al. (July 2010). "Gene therapy rescues cone function in congenital achromatopsia". Human Molecular Genetics. 19 (13): 2581–2593. doi:10.1093/hmg/ddq136. PMC 2883338. PMID 20378608.
Cavazzana-Calvo M, Payen E, Negre O, Wang G, Hehir K, Fusil F, et al. (September 2010). "Transfusion independence and HMGA2 activation after gene therapy of human β-thalassaemia". Nature. 467 (7313): 318–322. Bibcode:2010Natur.467..318C. doi:10.1038/nature09328. PMC 3355472. PMID 20844535.
Galanello R, Origa R (May 2010). "Beta-thalassemia". Orphanet Journal of Rare Diseases. 5 11. doi:10.1186/1750-1172-5-11. PMC 2893117. PMID 20492708.
Leboulch P (20 March 2013). "Five year outcome of lentiviral gene therapy for human beta-thalassemia, lessons and prospects". Thalassemia Reports. 3 (1s): 108. doi:10.4081/thal.2013.s1.e43.
Trojan A, Aristizabal BH, Jay LM, Castillo T, Penagos PJ, Briceño I, Trojan J (2016). "IGF-I biomarker testing in an ethical context". Adv Modern Oncol Res. 2 (4): 188–200. doi:10.18282/amor.v2.i4.58 (inactive 12 July 2025).{{cite journal}}: CS1 maint: DOI inactive as of July 2025 (link)
Castillo T, Trojan A, Noguera MC, Jay LM, Crane C, Alvarez A, et al. (2016). "Epistemológica experiencia en la elaboración de tecnología biomolecular para estrategia de la inmunoterapia génica" [Epistemological experience in developing of molecular biology technology for immunogene therapy strategy]. Rev Cien (in Spanish). 2 (25): 228–240. doi:10.14483//udistrital.jour.RC.2016.25.a6. doi:10.14483//udistrital.jour.RC.2016.25.a6
Yang ZJ, Zhang YR, Chen B, Zhang SL, Jia EZ, Wang LS, et al. (July 2009). "Phase I clinical trial on intracoronary administration of Ad-hHGF treating severe coronary artery disease". Molecular Biology Reports. 36 (6): 1323–1329. doi:10.1007/s11033-008-9315-3. PMID 18649012. S2CID 23419866.
Hahn W, Pyun WB, Kim DS, Yoo WS, Lee SD, Won JH, et al. (October 2011). "Enhanced cardioprotective effects by coexpression of two isoforms of hepatocyte growth factor from naked plasmid DNA in a rat ischemic heart disease model". The Journal of Gene Medicine. 13 (10): 549–555. doi:10.1002/jgm.1603. PMID 21898720. S2CID 26812780.
Deev RV, Bozo IY, Mzhavanadze ND, Voronov DA, Gavrilenko AV, Chervyakov YV, et al. (September 2015). "pCMV-vegf165 Intramuscular Gene Transfer is an Effective Method of Treatment for Patients With Chronic Lower Limb Ischemia". Journal of Cardiovascular Pharmacology and Therapeutics. 20 (5): 473–482. doi:10.1177/1074248415574336. PMID 25770117. S2CID 13443907.
Morrison C (3 March 2015). "$1-million price tag set for Glybera gene therapy". TradeSecrets. 33 (3): 217–218. doi:10.1038/nbt0315-217. PMID 25748892. S2CID 205266596.
Fernàndez-Ruiz I (March 2016). "Gene therapy: No improvement in outcomes with gene therapy for heart failure". Nature Reviews. Cardiology. 13 (3): 122–123. doi:10.1038/nrcardio.2016.14. PMID 26843287. S2CID 205472001.
Biffi A, Montini E, Lorioli L, Cesani M, Fumagalli F, Plati T, et al. (August 2013). "Lentiviral hematopoietic stem cell gene therapy benefits metachromatic leukodystrophy". Science. 341 (6148) 1233158. doi:10.1126/science.1233158. PMID 23845948. S2CID 206546808.
Aiuti A, Biasco L, Scaramuzza S, Ferrua F, Cicalese MP, Baricordi C, et al. (August 2013). "Lentiviral hematopoietic stem cell gene therapy in patients with Wiskott–Aldrich syndrome". Science. 341 (6148) 1233151. doi:10.1126/science.1233151. PMC 4375961. PMID 23845947.
Malech HL, Ochs HD (April 2015). "An emerging era of clinical benefit from gene therapy". JAMA. 313 (15): 1522–1523. doi:10.1001/jama.2015.2055. PMID 25898049.
Tebas P, Stein D, Tang WW, Frank I, Wang SQ, Lee G, et al. (March 2014). "Gene editing of CCR5 in autologous CD4 T cells of persons infected with HIV". The New England Journal of Medicine. 370 (10): 901–910. doi:10.1056/NEJMoa1300662. PMC 4084652. PMID 24597865.
Gardner MR, Kattenhorn LM, Kondur HR, von Schaewen M, Dorfman T, Chiang JJ, et al. (March 2015). "AAV-expressed eCD4-Ig provides durable protection from multiple SHIV challenges". Nature. 519 (7541): 87–91. Bibcode:2015Natur.519...87G. doi:10.1038/nature14264. PMC 4352131. PMID 25707797.
Cyranoski D (July 2016). "Chinese scientists to pioneer first human CRISPR trial". Nature. 535 (7613): 476–477. Bibcode:2016Natur.535..476C. doi:10.1038/nature.2016.20302. PMID 27466105.
Lee TW, Southern KW, Perry LA, Penny-Dimri JC, Aslam AA (June 2016). Southern KW (ed.). "Topical cystic fibrosis transmembrane conductance regulator gene replacement for cystic fibrosis-related lung disease". The Cochrane Database of Systematic Reviews. 2016 (6) CD005599. doi:10.1002/14651858.CD005599.pub5. PMC 8682957. PMID 27314455.
Kaiser J (November 2017). "A human has been injected with gene-editing tools to cure his disabling disease. Here's what you need to know". Science. doi:10.1126/science.aar5098.
Ledford H (5 September 2018). "First test of in-body gene editing shows promise". Nature. doi:10.1038/d41586-018-06195-6. S2CID 92840885.
Rangarajan S, Walsh L, Lester W, Perry D, Madan B, Laffan M, et al. (December 2017). "AAV5-Factor VIII Gene Transfer in Severe Hemophilia A". The New England Journal of Medicine. 377 (26): 2519–2530. doi:10.1056/nejmoa1708483. hdl:10044/1/57163. PMID 29224506.
van den Berg HM (December 2017). "A Cure for Hemophilia within Reach". The New England Journal of Medicine. 377 (26): 2592–2593. doi:10.1056/nejme1713888. PMID 29224412.
Ledford H (March 2020). "CRISPR treatment inserted directly into the body for first time". Nature. 579 (7798): 185. Bibcode:2020Natur.579..185L. doi:10.1038/d41586-020-00655-8. PMID 32157225.
"High-dose AAV gene therapy deaths". Nature Biotechnology. 38 (8): 910. 1 August 2020. doi:10.1038/s41587-020-0642-9. ISSN 1546-1696. PMID 32760031. S2CID 220981004.
Kohn DB, Booth C, Shaw KL, Xu-Bayford J, Garabedian E, Trevisan V, et al. (May 2021). "Autologous Ex Vivo Lentiviral Gene Therapy for Adenosine Deaminase Deficiency". The New England Journal of Medicine. 384 (21): 2002–2013. doi:10.1056/NEJMoa2027675. PMC 8240285. PMID 33974366.
Mamcarz E, Zhou S, Lockey T, Abdelsamed H, Cross SJ, Kang G, et al. (April 2019). "Lentiviral Gene Therapy Combined with Low-Dose Busulfan in Infants with SCID-X1". The New England Journal of Medicine. 380 (16): 1525–1534. doi:10.1056/NEJMoa1815408. PMC 6636624. PMID 30995372.
Gillmore JD, Gane E, Taubel J, Kao J, Fontana M, Maitland ML, et al. (August 2021). "CRISPR-Cas9 In Vivo Gene Editing for Transthyretin Amyloidosis". The New England Journal of Medicine. 385 (6): 493–502. doi:10.1056/NEJMoa2107454. PMID 34215024. S2CID 235722446.
"The science news that shaped 2021: Nature's picks". Nature. December 2021. doi:10.1038/d41586-021-03734-6. PMID 34907370. S2CID 245144020.
Pearson TS, Gupta N, San Sebastian W, Imamura-Ching J, Viehoever A, Grijalvo-Perez A, et al. (July 2021). "Gene therapy for aromatic L-amino acid decarboxylase deficiency by MR-guided direct delivery of AAV2-AADC to midbrain dopaminergic neurons". Nature Communications. 12 (1) 4251. Bibcode:2021NatCo..12.4251P. doi:10.1038/s41467-021-24524-8. PMC 8275582. PMID 34253733.
Flotte TR, Cataltepe O, Puri A, Batista AR, Moser R, McKenna-Yasek D, et al. (February 2022). "AAV gene therapy for Tay-Sachs disease". Nature Medicine. 28 (2): 251–259. doi:10.1038/s41591-021-01664-4. PMC 10786171. PMID 35145305. S2CID 246748772.
Chowdary P, Shapiro S, Makris M, Evans G, Boyce S, Talks K, et al. (July 2022). "Phase 1-2 Trial of AAVS3 Gene Therapy in Patients with Hemophilia B". The New England Journal of Medicine. 387 (3): 237–247. doi:10.1056/NEJMoa2119913. PMID 35857660. S2CID 250697905.
Huang, Jinbo; Xue, Shuai; Buchmann, Peter; Teixeira, Ana Palma; Fussenegger, Martin (16 August 2023). "An electrogenetic interface to program mammalian gene expression by direct current". Nature Metabolism. 5 (8): 1395–1407. doi:10.1038/s42255-023-00850-7. PMC 10447240. PMID 37524785.

editasmedicine.com (Global: low place; English: low place)

ir.editasmedicine.com

"Allergan and Editas Medicine Initiate the Brilliance Phase 1/2 Clinical Trial of AGN-151587 (EDIT-101) for the Treatment of LCA10". Editas Medicine. Retrieved 20 August 2019.

endpts.com (Global: low place; English: low place)

Tong A (3 June 2019). "EU stamps historic OK on bluebird's gene therapy for β-thalassemia – now sit back and wait for the price". Retrieved 4 June 2019.

eurekalert.org (Global: 3,538^th place; English: 3,083^rd place)

"Penn Medicine presents HIV gene therapy trial data at CROI 2009". EurekAlert!. 10 February 2009. Archived from the original on 25 August 2012. Retrieved 19 November 2009.

eurolab.ua (Global: low place; English: low place)

"Neovasculogen". Eurolab. Retrieved 4 August 2015.

europa.eu (Global: 68^th place; English: 117^th place)

ema.europa.eu

"Tecartus". European Medicines Agency. 13 October 2020.
"Upstaza EPAR". European Medicines Agency (EMA). 13 April 2022. Archived from the original on 14 March 2023. Retrieved 3 March 2023. Text was copied from this source which is copyright European Medicines Agency. Reproduction is authorized provided the source is acknowledged.
"Imlygic". European Medicines Agency (EMA). 4 June 2018.
"First gene therapy to treat severe haemophilia A". European Medicines Agency (EMA) (Press release). 24 June 2022. Archived from the original on 26 June 2022. Retrieved 26 June 2022.
"Roctavian: Pending EC decision". European Medicines Agency (EMA). 23 June 2022. Archived from the original on 26 June 2022. Retrieved 26 June 2022.
"Luxturna". European Medicines Agency (EMA). 24 September 2018.
"Summary of opinion1 (initial authorisation) Strimvelis" (PDF). European Medicines Agency. 1 April 2016. pp. 1–2. Archived from the original (PDF) on 28 January 2018. Retrieved 13 April 2016.
"Zynteglo EPAR". European Medicines Agency (EMA). 25 March 2019. Retrieved 8 June 2020. Text was copied from this source which is copyright European Medicines Agency. Reproduction is authorized provided the source is acknowledged.
"Zolgensma EPAR". European Medicines Agency (EMA). 24 March 2020. Retrieved 16 October 2020.
"Libmeldy: Pending EC decision". European Medicines Agency (EMA). 16 October 2020. Archived from the original on 17 October 2020. Retrieved 16 October 2020. Text was copied from this source which is copyright European Medicines Agency. Reproduction is authorized provided the source is acknowledged.
"New gene therapy to treat rare genetic disorder metachromatic leukodystrophy". European Medicines Agency. 16 October 2020. Retrieved 16 October 2020. Text was copied from this source which is copyright European Medicines Agency. Reproduction is authorized provided the source is acknowledged.
"Libmeldy EPAR". European Medicines Agency (EMA). 13 October 2020. Retrieved 22 December 2020.
"Upstaza: Pending EC decision". European Medicines Agency. 19 May 2022. Archived from the original on 25 May 2022. Retrieved 22 May 2022.

ec.europa.eu

"Roctavian". Union Register of medicinal products. 25 August 2022. Retrieved 6 September 2022.

factcheck.org (Global: 7,075^th place; English: 4,103^rd place)

Spencer SH (28 June 2022). "Website Peddles Old, Debunked Falsehood About COVID-19 mRNA Vaccines". FactCheck.org. Retrieved 19 February 2023.

fda.gov (Global: 447^th place; English: 338^th place)

"What is Gene Therapy?". U.S. Food and Drug Administration (FDA). 9 December 2020. Archived from the original on 7 June 2019.
"FDA grants accelerated approval to afamitresgene autoleucel for unresectable or metastatic synovial sarcoma". U.S. Food and Drug Administration (FDA). 2 August 2024. Retrieved 19 September 2024. This article incorporates text from this source, which is in the public domain.
"FDA Approves First Gene Therapy for Children with Metachromatic Leukodystrophy". U.S. Food and Drug Administration (FDA) (Press release). 9 August 2024. Archived from the original on 18 March 2024.
"FDA Approves First Topical Gene Therapy for Treatment of Wounds in Patients with Dystrophic Epidermolysis Bullosa". U.S. Food and Drug Administration (FDA) (Press release). 19 May 2023. Archived from the original on 19 May 2023. Retrieved 31 July 2023.
"FDA Approves First Cell-Based Gene Therapy to Treat Adult and Pediatric Patients with Beta-thalassemia Who Require Regular Blood Transfusions". U.S. Food and Drug Administration (FDA) (Press release). 17 August 2022. Archived from the original on 17 August 2022. Retrieved 31 August 2022.
"FDA Approves First Cell-Based Gene Therapy For Adult Patients with Relapsed or Refractory MCL". U.S. Food and Drug Administration (FDA) (Press release). 24 July 2020. Retrieved 24 July 2020.^{[dead link]}
"FDA approves brexucabtagene autoleucel for relapsed or refractory B-cell precursor acute lymphoblastic leukemia". U.S. Food and Drug Administration (FDA). 1 October 2021. Archived from the original on 2 October 2021. Retrieved 2 October 2021.
"FDA Approves First Gene Therapy for Treatment of Certain Patients with Duchenne Muscular Dystrophy". U.S. Food and Drug Administration (FDA) (Press release). 23 June 2023. Archived from the original on 22 June 2023. Retrieved 12 July 2023.
"FDA Approves First Gene Therapy for Treatment of Aromatic L-amino Acid Decarboxylase Deficiency". U.S. Food and Drug Administration (FDA) (Press release). 14 November 2024. Archived from the original on 14 November 2024. Retrieved 17 November 2024. This article incorporates text from this source, which is in the public domain.
"Casgevy". U.S. Food and Drug Administration (FDA). 8 December 2023. Archived from the original on 8 December 2023. Retrieved 8 December 2023.
"Abecma (idecabtagene vicleucel)". U.S. Food and Drug Administration (FDA). 21 April 2021. Archived from the original on 17 May 2021.
"Breyanzi (lisocabtagene maraleucel)". U.S. Food and Drug Administration (FDA). 4 March 2021. Archived from the original on 28 February 2021.
"Lyfgenia". U.S. Food and Drug Administration (FDA). 8 December 2023. Archived from the original on 8 December 2023. Retrieved 9 December 2023.
"FDA Approves First Gene Therapy for the Treatment of High-Risk, Non-Muscle-Invasive Bladder Cancer". U.S. Food and Drug Administration (FDA) (Press release). 16 December 2022. Archived from the original on 16 December 2022. Retrieved 16 December 2022.
"FDA approves obecabtagene autoleucel for acute lymphoblastic leukemia". U.S. Food and Drug Administration (FDA). 8 November 2024. Archived from the original on 8 November 2024. Retrieved 10 November 2024. This article incorporates text from this source, which is in the public domain.
"Zolgensma". U.S. Food and Drug Administration (FDA). 16 March 2021. Archived from the original on 12 September 2019.
"Zevaskyn". U.S. Food and Drug Administration (FDA). 28 April 2025. Retrieved 7 July 2025.
"Encelto". U.S. Food and Drug Administration. 1 October 2024. Retrieved 7 March 2025.
"FDA approves novel gene therapy to treat patients with a rare form of inherited vision loss". U.S. Food and Drug Administration (FDA) (Press release). 24 March 2020. Archived from the original on 26 May 2019. Retrieved 23 July 2021.
U.S. Food and Drug Administration (14 October 1993). "Application of Current Statutory Authorities to Human Somatic Cell Therapy Products and Gene Therapy Products" (PDF). Federal Register. 58 (197). Archived from the original (PDF) on 21 June 2009.
"Guidance for Industry: Guidance for Human Somatic Cell Therapy and Gene Therapy". U.S. Food and Drug Administration (FDA). 9 July 2009. Archived from the original on 9 July 2009. Retrieved 2 January 2022.
"FDA approval brings first gene therapy to the United States" (Press release). U.S. Food and Drug Administration (FDA). 30 August 2017. Archived from the original on 13 May 2019.
"FDA approves axicabtagene ciloleucel for large B-cell lymphoma". U.S. Food and Drug Administration (FDA). Archived from the original on 12 June 2019. Retrieved 5 January 2018.
"FDA approves novel gene therapy to treat patients with a rare form of inherited vision loss". U.S. Food and Drug Administration (FDA) (Press release). 19 December 2017. Archived from the original on 26 May 2019. Retrieved 20 December 2017.

fiercebiotech.com (Global: low place; English: 8,828^th place)

"Astellas' Audentes reports 3rd death in gene therapy trial". Fierce Biotech. 21 August 2020. Retrieved 21 September 2020.

forbes.com (Global: 54^th place; English: 48^th place)

Herper M (26 March 2014). "Gene Therapy's Big Comeback". Forbes. Retrieved 28 April 2014.
Herper M (2 January 2018). "Spark Therapeutics Sets Price Of Blindness-Treating Gene Therapy At $850,000". Forbes. Retrieved 4 January 2018.

fullfact.org (Global: low place; English: low place)

"Andrew Bridgen wrong to call mRNA vaccines gene therapy". Full Fact. 12 January 2023. Retrieved 19 February 2023.
"Andrew Bridgen MP's false claims put lives at risk". Full Fact. 11 January 2023. Retrieved 19 February 2023.

genome.gov (Global: 9,308^th place; English: 8,899^th place)

Hanna K (2006). "Germline Gene Transfer". National Human Genome Research Institute.

ghostarchive.org (Global: 32^nd place; English: 21^st place)

Zimmer C (16 September 2013). "DNA Double Take". The New York Times. Archived from the original on 2 January 2022.
Wade N (19 March 2015). "Scientists Seek Ban on Method of Editing the Human Genome". The New York Times. Archived from the original on 2 January 2022. Retrieved 20 March 2015.
Pollack A (3 March 2015). "A Powerful New Way to Edit DNA". The New York Times. Archived from the original on 2 January 2022. Retrieved 20 March 2015.
Kolata G (23 April 2015). "Chinese Scientists Edit Genes of Human Embryos, Raising Concerns". The New York Times. Archived from the original on 2 January 2022. Retrieved 24 April 2015.
Harmon A (14 February 2017). "Human Gene Editing Receives Science Panel's Support". The New York Times. ISSN 0362-4331. Archived from the original on 2 January 2022. Retrieved 17 February 2017.
Zimmer C (9 March 2015). "Protection Without a Vaccine". The New York Times. Archived from the original on 2 January 2022. Retrieved 30 March 2015.
Wade N (3 December 2015). "Scientists Place Moratorium on Edits to Human Genome That Could Be Inherited". The New York Times. Archived from the original on 2 January 2022. Retrieved 3 December 2015.

globenewswire.com (Global: 2,614^th place; English: 1,549^th place)

"Orchard Therapeutics Receives EC Approval for Libmeldy for the Treatment of Early-Onset Metachromatic Leukodystrophy (MLD)" (Press release). Orchard Therapeutics. 21 December 2020. Retrieved 23 July 2021 – via GlobeNewswire.
"Autolus Therapeutics Announces FDA Approval of Aucatzyl (obecabtagene autoleucel – obe-cel) for adults with relapsed/refractory B-cell acute lymphoblastic leukemia (r/r B-ALL)" (Press release). Autolus Therapeutics. 8 November 2024. Retrieved 10 November 2024 – via GlobeNewswire.

handle.net (Global: 102^nd place; English: 76^th place)

hdl.handle.net

Bainbridge JW, Smith AJ, Barker SS, Robbie S, Henderson R, Balaggan K, et al. (May 2008). "Effect of gene therapy on visual function in Leber's congenital amaurosis". The New England Journal of Medicine. 358 (21): 2231–2239. doi:10.1056/NEJMoa0802268. hdl:10261/271174. PMID 18441371.
Cavazzana-Calvo M, Thrasher A, Mavilio F (February 2004). "The future of gene therapy". Nature. 427 (6977): 779–781. Bibcode:2004Natur.427..779C. doi:10.1038/427779a. hdl:11380/307566. PMID 14985734. S2CID 4421364.
Rangarajan S, Walsh L, Lester W, Perry D, Madan B, Laffan M, et al. (December 2017). "AAV5-Factor VIII Gene Transfer in Severe Hemophilia A". The New England Journal of Medicine. 377 (26): 2519–2530. doi:10.1056/nejmoa1708483. hdl:10044/1/57163. PMID 29224506.

harvard.edu (Global: 18^th place; English: 17^th place)

ui.adsabs.harvard.edu

Sun M (October 1982). "Martin Cline loses appeal on NIH grant". Science. 218 (4567): 37. Bibcode:1982Sci...218...37S. doi:10.1126/science.7123214. PMID 7123214.
Dever DP, Bak RO, Reinisch A, Camarena J, Washington G, Nicolas CE, et al. (November 2016). "CRISPR/Cas9 β-globin gene targeting in human haematopoietic stem cells". Nature. 539 (7629): 384–389. Bibcode:2016Natur.539..384D. doi:10.1038/nature20134. PMC 5898607. PMID 27820943.
Gupta RM, Musunuru K (October 2014). "Expanding the genetic editing tool kit: ZFNs, TALENs, and CRISPR-Cas9". The Journal of Clinical Investigation. 124 (10): 4154–61. Bibcode:2014JCliI.124.4154G. doi:10.1172/JCI72992. PMC 4191047. PMID 25271723.
Bertrand N, Grenier P, Mahmoudi M, Lima EM, Appel EA, Dormont F, et al. (October 2017). "Mechanistic understanding of in vivo protein corona formation on polymeric nanoparticles and impact on pharmacokinetics". Nature Communications. 8 (1) 777. Bibcode:2017NatCo...8..777B. doi:10.1038/s41467-017-00600-w. PMC 5626760. PMID 28974673.
Woods NB, Bottero V, Schmidt M, von Kalle C, Verma IM (April 2006). "Gene therapy: therapeutic gene causing lymphoma". Nature. 440 (7088): 1123. Bibcode:2006Natur.440.1123W. doi:10.1038/4401123a. PMID 16641981. S2CID 4372110.
Thrasher AJ, Gaspar HB, Baum C, Modlich U, Schambach A, Candotti F, et al. (September 2006). "Gene therapy: X-SCID transgene leukaemogenicity". Nature. 443 (7109): E5-6, discussion E6-7. Bibcode:2006Natur.443E...5T. doi:10.1038/nature05219. PMID 16988659. S2CID 4301764.
Roco MC, Bainbridge WS (2002). "Converging Technologies for Improving Human Performance: Integrating From the Nanoscale". Journal of Nanoparticle Research. 4 (4): 281–295. Bibcode:2002JNR.....4..281R. doi:10.1023/A:1021152023349. S2CID 136290217.
Baltimore D, Berg P, Botchan M, Carroll D, Charo RA, Church G, et al. (April 2015). "Biotechnology. A prudent path forward for genomic engineering and germline gene modification". Science. 348 (6230): 36–38. Bibcode:2015Sci...348...36B. doi:10.1126/science.aab1028. PMC 4394183. PMID 25791083.
Lanphier E, Urnov F, Haecker SE, Werner M, Smolenski J (March 2015). "Don't edit the human germ line". Nature. 519 (7544): 410–411. Bibcode:2015Natur.519..410L. doi:10.1038/519410a. PMID 25810189.
Friedmann T, Roblin R (March 1972). "Gene therapy for human genetic disease?". Science. 175 (4025): 949–955. Bibcode:1972Sci...175..949F. doi:10.1126/science.175.4025.949. PMID 5061866. S2CID 19952096.
Blaese RM, Culver KW, Miller AD, Carter CS, Fleisher T, Clerici M, et al. (October 1995). "T lymphocyte-directed gene therapy for ADA- SCID: initial trial results after 4 years". Science. 270 (5235): 475–480. Bibcode:1995Sci...270..475B. doi:10.1126/science.270.5235.475. PMID 7570001. S2CID 46339645.
Trojan J, Johnson TR, Rudin SD, Ilan J, Tykocinski ML, Ilan J (January 1993). "Treatment and prevention of rat glioblastoma by immunogenic C6 cells expressing antisense insulin-like growth factor I RNA". Science. 259 (5091): 94–97. Bibcode:1993Sci...259...94T. doi:10.1126/science.8418502. PMID 8418502.
Abbott A (April 1992). "Gene therapy. Italians first to use stem cells". Nature. 356 (6369): 465. Bibcode:1992Natur.356..465A. doi:10.1038/356465a0. PMID 1560817. S2CID 4319842.
Cavazzana-Calvo M, Thrasher A, Mavilio F (February 2004). "The future of gene therapy". Nature. 427 (6977): 779–781. Bibcode:2004Natur.427..779C. doi:10.1038/427779a. hdl:11380/307566. PMID 14985734. S2CID 4421364.
Naldini, Luigi; Blömer, Ulrike; Gallay, Philippe; Ory, Daniel; Mulligan, Richard; Gage, Fred H.; Verma, Inder M.; Trono, Didier (12 April 1996). "In Vivo Gene Delivery and Stable Transduction of Nondividing Cells by a Lentiviral Vector". Science. 272 (5259): 263–267. Bibcode:1996Sci...272..263N. doi:10.1126/science.272.5259.263. ISSN 0036-8075. PMID 8602510. S2CID 18997464.
Morgan RA, Dudley ME, Wunderlich JR, Hughes MS, Yang JC, Sherry RM, et al. (October 2006). "Cancer regression in patients after transfer of genetically engineered lymphocytes". Science. 314 (5796): 126–129. Bibcode:2006Sci...314..126M. doi:10.1126/science.1129003. PMC 2267026. PMID 16946036.
Levine BL, Humeau LM, Boyer J, MacGregor RR, Rebello T, Lu X, et al. (November 2006). "Gene transfer in humans using a conditionally replicating lentiviral vector". Proceedings of the National Academy of Sciences of the United States of America. 103 (46): 17372–17377. Bibcode:2006PNAS..10317372L. doi:10.1073/pnas.0608138103. PMC 1635018. PMID 17090675.
Cartier N, Hacein-Bey-Abina S, Bartholomae CC, Veres G, Schmidt M, Kutschera I, et al. (November 2009). "Hematopoietic stem cell gene therapy with a lentiviral vector in X-linked adrenoleukodystrophy" (PDF). Science. 326 (5954): 818–823. Bibcode:2009Sci...326..818C. doi:10.1126/science.1171242. PMID 19892975. S2CID 27783.
Cavazzana-Calvo M, Payen E, Negre O, Wang G, Hehir K, Fusil F, et al. (September 2010). "Transfusion independence and HMGA2 activation after gene therapy of human β-thalassaemia". Nature. 467 (7313): 318–322. Bibcode:2010Natur.467..318C. doi:10.1038/nature09328. PMC 3355472. PMID 20844535.
Gardner MR, Kattenhorn LM, Kondur HR, von Schaewen M, Dorfman T, Chiang JJ, et al. (March 2015). "AAV-expressed eCD4-Ig provides durable protection from multiple SHIV challenges". Nature. 519 (7541): 87–91. Bibcode:2015Natur.519...87G. doi:10.1038/nature14264. PMC 4352131. PMID 25707797.
Cyranoski D (July 2016). "Chinese scientists to pioneer first human CRISPR trial". Nature. 535 (7613): 476–477. Bibcode:2016Natur.535..476C. doi:10.1038/nature.2016.20302. PMID 27466105.
Ledford H (March 2020). "CRISPR treatment inserted directly into the body for first time". Nature. 579 (7798): 185. Bibcode:2020Natur.579..185L. doi:10.1038/d41586-020-00655-8. PMID 32157225.
Pearson TS, Gupta N, San Sebastian W, Imamura-Ching J, Viehoever A, Grijalvo-Perez A, et al. (July 2021). "Gene therapy for aromatic L-amino acid decarboxylase deficiency by MR-guided direct delivery of AAV2-AADC to midbrain dopaminergic neurons". Nature Communications. 12 (1) 4251. Bibcode:2021NatCo..12.4251P. doi:10.1038/s41467-021-24524-8. PMC 8275582. PMID 34253733.

hhs.gov (Global: 5,973^rd place; English: 3,693^rd place)

"Read the Belmont Report". HHS.gov. 15 January 2018. Retrieved 2 January 2022.

hugo-international.org (Global: low place; English: low place)

HUGO Ethics Committee (April 2001). "Statement on Gene Therapy Research" (PDF). Human Genome Organization. Archived from the original (PDF) on 7 September 2008.

independent.co.uk (Global: 36^th place; English: 33^rd place)

"These experts say Joe Rogan is 'extraordinarily dangerous' to society – here's why". The Independent. 2 February 2022. Retrieved 19 February 2023.
"Parents spark breakthrough gene therapy for children with Tay-Sachs disease". The Independent. 18 February 2022. Retrieved 7 March 2022.

io9.com (Global: 2,391^st place; English: 1,563^rd place)

Dvorsky G (6 March 2014). "Scientists Create Genetically Modified Cells That Protect Against HIV". io9, Biotechnology. Retrieved 6 March 2014.

jetpress.org (Global: low place; English: low place)

Allhoff F (March 2005). "Germ-line genetic enhancement and Rawlsian primary goods" (PDF). Kennedy Institute of Ethics Journal. 15 (1): 39–56. CiteSeerX 10.1.1.566.171. doi:10.1353/ken.2005.0007. PMID 15881795. S2CID 14432440. Archived (PDF) from the original on 30 January 2012.

josiahzayner.com (Global: low place; English: low place)

Zayner J. "The First Attempt At Human CRISPR Gene Editing". Retrieved 20 May 2020.

jyi.org (Global: low place; English: low place)

"Evaluation of the Clinical Success of Ex Vivo and In Vivo Gene Therapy". Journal of Young Investigators. January 2009. Retrieved 23 July 2021.

legacy.com (Global: 672^nd place; English: 400^th place)

"Lorraine Kraus Obituary". The Commercial Appeal. Retrieved 7 July 2023.

liebertpub.com (Global: low place; English: low place)

Asaleh A, Zhou J, Rahmanian N (November 2022). "Letter to the Editor: A Lesson for the Future-How Semantic Ambiguity Led to the Spread of Anti-Scientific Attitudes About COVID-19 mRNA Vaccines". Human Gene Therapy. 33 (21–22): 1213–1216. doi:10.1089/hum.2022.29223.aas (inactive 12 July 2025). PMID 36375123.{{cite journal}}: CS1 maint: DOI inactive as of July 2025 (link)

lifesciencesfoundation.org (Global: low place; English: low place)

"The first gene therapy". Life Sciences Foundation. 21 June 2011. Archived from the original on 28 November 2012. Retrieved 7 January 2014.

luxturna.com (Global: low place; English: low place)

"What is LUXTURNA?". LUXTURNA® (voretigene neparvovec-rzyl). Retrieved 17 November 2025.

lysogene.com (Global: low place; English: low place)

Lysogene (15 October 2020). "Lysogene provides update on the AAVance Clinical Trial Evaluating LYS-SAF302 in Patients with MPS IIIA – Lysogene". Archived from the original on 17 October 2020. Retrieved 17 October 2020.

medlineplus.gov (Global: 1,581^st place; English: 1,299^th place)

"What is gene therapy?". Genetics Home Reference. 28 March 2016. Archived from the original on 6 April 2016. Retrieved 2 January 2022.
"How does gene therapy work?". Genomics Home Reference. U.S. National Library of Medicine.

medpagetoday.com (Global: 9,274^th place; English: 5,856^th place)

"Death Prompts FDA to Suspend Arthritis Gene Therapy Trial". Medpage Today. 27 July 2007. Retrieved 10 November 2010.

medscape.com (Global: 719^th place; English: 636^th place)

Ott MG, Schmidt M, Schwarzwaelder K, Stein S, Siler U, Koehl U, et al. (April 2006). "Correction of X-linked chronic granulomatous disease by gene therapy, augmented by insertional activation of MDS1-EVI1, PRDM16 or SETBP1". Nature Medicine. 12 (4): 401–409. doi:10.1038/nm1393. PMID 16582916. S2CID 7601162.
Beals JK (16 September 2010). "Gene Therapy Frees Beta-Thalassemia Patient From Transfusions for 2+ Years". MedScape. Retrieved 15 December 2012.

missouri.edu (Global: 3,342^nd place; English: 3,036^th place)

ethics.missouri.edu

"The Center for Health Ethics - University of Missouri School of Medicine - Gene Therapy and Genetic Engineering". ethics.missouri.edu. 3 December 2013. Archived from the original on 3 December 2013. Retrieved 2 January 2022.

mskcc.org (Global: low place; English: 7,046^th place)

"On Cancer: Launch of Stem Cell Therapy Trial Offers Hope for Patients with Inherited Blood Disorder". Memorial Sloan Kettering Cancer Center. Memorial Sloan-Kettering Cancer Center. 16 July 2012. Archived from the original on 8 June 2020. Retrieved 15 December 2012.

nationalacademies.org (Global: 7,189^th place; English: 6,078^th place)

Committee on Human Gene Editing: Scientific, Medical, and Ethical Considerations . "Human Genome Editing: Science, Ethics, and Governance". nationalacademies.org. National Academy of Sciences; National Academy of Medicine. Retrieved 21 February 2017.

nature.com (Global: 234^th place; English: 397^th place)

Reardon S (14 February 2017). "US science advisers outline path to genetically modified babies". Nature. doi:10.1038/nature.2017.21474.

newscientist.com (Global: 774^th place; English: 716^th place)

Geddes L (30 October 2013). "'Bubble kid' success puts gene therapy back on track". New Scientist. Retrieved 2 January 2022.
Coghlan A (26 March 2013). "Gene therapy cures leukaemia in eight days". The New Scientist. Retrieved 15 April 2013.
Coghlan A (11 December 2013). "Souped-up immune cells force leukaemia into remission". New Scientist. Retrieved 15 April 2013.
"Gene therapy needs a hero to live up to the hype". New Scientist. 30 October 2013. Retrieved 2 January 2022.
Penman D (11 October 2002). "Subtle gene therapy tackles blood disorder". New Scientist. Retrieved 17 August 2010.
"DNA nanoballs boost gene therapy". New Scientist. 12 May 2002. Retrieved 17 August 2010.
Ananthaswamy A (20 March 2003). "Undercover genes slip into the brain". New Scientist. Retrieved 17 August 2010.
Holmes R (13 March 2003). "Gene therapy may switch off Huntington's". New Scientist. Retrieved 17 August 2010.
Beali A (25 January 2014). "Gene therapy restores sight in people with eye disease". The New Scientist. Retrieved 25 January 2014.
Coghlan A (9 April 2016). "Gene Therapy Approved". New Scientist. No. 3068. pp. 8–9.
Coghlan A (March 2017). "Gene therapy 'cures' boy of blood disease that affects millions". New Scientist.

newsweek.com (Global: 269^th place; English: 201^st place)

Sheridan K (19 December 2017). "The FDA approved a gene therapy to treat blindness in a groundbreaking moment for DNA-based medicine". Newsweek. Retrieved 20 December 2017.

nih.gov (Global: 4^th place; English: 4^th place)

pubmed.ncbi.nlm.nih.gov

Kaji EH, Leiden JM (February 2001). "Gene and stem cell therapies". JAMA. 285 (5): 545–550. doi:10.1001/jama.285.5.545. PMID 11176856.
Rosenberg SA, Aebersold P, Cornetta K, Kasid A, Morgan RA, Moen R, et al. (August 1990). "Gene transfer into humans--immunotherapy of patients with advanced melanoma, using tumor-infiltrating lymphocytes modified by retroviral gene transduction". The New England Journal of Medicine. 323 (9): 570–578. doi:10.1056/NEJM199008303230904. PMID 2381442.
Gorell E, Nguyen N, Lane A, Siprashvili Z (April 2014). "Gene therapy for skin diseases". Cold Spring Harbor Perspectives in Medicine. 4 (4) a015149. doi:10.1101/cshperspect.a015149. PMC 3968787. PMID 24692191.
Sun M (October 1982). "Martin Cline loses appeal on NIH grant". Science. 218 (4567): 37. Bibcode:1982Sci...218...37S. doi:10.1126/science.7123214. PMID 7123214.
Sheridan C (February 2011). "Gene therapy finds its niche". Nature Biotechnology. 29 (2): 121–128. doi:10.1038/nbt.1769. PMID 21301435. S2CID 5063701.
O'Malley BW, Ledley FD (October 1993). "Somatic gene therapy. Methods for the present and future". Arch Otolaryngol Head Neck Surg. 119 (10): 1100–7. doi:10.1001/archotol.1993.01880220044007. PMID 8398061.
Oldfield EH, Ram Z, Culver KW, Blaese RM, DeVroom HL, Anderson WF (February 1993). "Gene therapy for the treatment of brain tumors using intra-tumoral transduction with the thymidine kinase gene and intravenous ganciclovir". Human Gene Therapy. 4 (1): 39–69. doi:10.1089/hum.1993.4.1-39. PMID 8384892.
Maguire AM, Simonelli F, Pierce EA, Pugh EN, Mingozzi F, Bennicelli J, et al. (May 2008). "Safety and efficacy of gene transfer for Leber's congenital amaurosis". The New England Journal of Medicine. 358 (21): 2240–2248. doi:10.1056/NEJMoa0802315. PMC 2829748. PMID 18441370.
Simonelli F, Maguire AM, Testa F, Pierce EA, Mingozzi F, Bennicelli JL, et al. (March 2010). "Gene therapy for Leber's congenital amaurosis is safe and effective through 1.5 years after vector administration". Molecular Therapy. 18 (3): 643–650. doi:10.1038/mt.2009.277. PMC 2839440. PMID 19953081.
Cideciyan AV, Hauswirth WW, Aleman TS, Kaushal S, Schwartz SB, Boye SL, Windsor EA, Conlon TJ, Sumaroka A, Roman AJ, Byrne BJ, Jacobson SG (August 2009). "Vision 1 year after gene therapy for Leber's congenital amaurosis". The New England Journal of Medicine. 361 (7): 725–727. doi:10.1056/NEJMc0903652. PMC 2847775. PMID 19675341.
Bainbridge JW, Smith AJ, Barker SS, Robbie S, Henderson R, Balaggan K, et al. (May 2008). "Effect of gene therapy on visual function in Leber's congenital amaurosis". The New England Journal of Medicine. 358 (21): 2231–2239. doi:10.1056/NEJMoa0802268. hdl:10261/271174. PMID 18441371.
Fischer A, Hacein-Bey-Abina S, Cavazzana-Calvo M (June 2010). "20 years of gene therapy for SCID". Nature Immunology. 11 (6): 457–460. doi:10.1038/ni0610-457. PMID 20485269. S2CID 11300348.
Ferrua F, Brigida I, Aiuti A (December 2010). "Update on gene therapy for adenosine deaminase-deficient severe combined immunodeficiency". Current Opinion in Allergy and Clinical Immunology. 10 (6): 551–556. doi:10.1097/ACI.0b013e32833fea85. PMID 20966749. S2CID 205435278.
Cartier N, Aubourg P (July 2010). "Hematopoietic stem cell transplantation and hematopoietic stem cell gene therapy in X-linked adrenoleukodystrophy". Brain Pathology. 20 (4): 857–862. doi:10.1111/j.1750-3639.2010.00394.x. PMC 8094635. PMID 20626747. S2CID 24182017.
LeWitt PA, Rezai AR, Leehey MA, Ojemann SG, Flaherty AW, Eskandar EN, et al. (April 2011). "AAV2-GAD gene therapy for advanced Parkinson's disease: a double-blind, sham-surgery controlled, randomised trial". The Lancet. Neurology. 10 (4): 309–319. doi:10.1016/S1474-4422(11)70039-4. PMID 21419704. S2CID 37154043.
Pearson S, Jia H, Kandachi K (January 2004). "China approves first gene therapy". Nature Biotechnology. 22 (1): 3–4. doi:10.1038/nbt0104-3. PMC 7097065. PMID 14704685.
Pezzoli D, Chiesa R, De Nardo L, Candiani G (September 2012). "We still have a long way to go to effectively deliver genes!". Journal of Applied Biomaterials & Functional Materials. 10 (2): 82–91. doi:10.5301/JABFM.2012.9707. PMID 23015375. S2CID 6283455.
Vannucci L, Lai M, Chiuppesi F, Ceccherini-Nelli L, Pistello M (January 2013). "Viral vectors: a look back and ahead on gene transfer technology". The New Microbiologica. 36 (1): 1–22. PMID 23435812.
Gothelf A, Gehl J (November 2012). "What you always needed to know about electroporation based DNA vaccines". Human Vaccines & Immunotherapeutics. 8 (11): 1694–1702. doi:10.4161/hv.22062. PMC 3601144. PMID 23111168.
Urnov FD, Rebar EJ, Holmes MC, Zhang HS, Gregory PD (September 2010). "Genome editing with engineered zinc finger nucleases". Nature Reviews Genetics. 11 (9): 636–646. doi:10.1038/nrg2842. PMID 20717154. S2CID 205484701.
Bak RO, Gomez-Ospina N, Porteus MH (August 2018). "Gene Editing on Center Stage". Trends in Genetics. 34 (8): 600–611. doi:10.1016/j.tig.2018.05.004. PMID 29908711. S2CID 49269023.
Stone D, Niyonzima N, Jerome KR (September 2016). "Genome editing and the next generation of antiviral therapy". Human Genetics. 135 (9): 1071–82. doi:10.1007/s00439-016-1686-2. PMC 5002242. PMID 27272125.
Cross D, Burmester JK (September 2006). "Gene therapy for cancer treatment: past, present and future". Clinical Medicine & Research. 4 (3): 218–27. doi:10.3121/cmr.4.3.218. PMC 1570487. PMID 16988102.
Maeder ML, Gersbach CA (March 2016). "Genome-editing Technologies for Gene and Cell Therapy". Molecular Therapy. 24 (3): 430–46. doi:10.1038/mt.2016.10. PMC 4786923. PMID 26755333.
"First CRISPR therapy dosed". Nature Biotechnology. 38 (4): 382. 1 April 2020. doi:10.1038/s41587-020-0493-4. ISSN 1546-1696. PMID 32265555. S2CID 215406440.
Sherkow JS, Zettler PJ, Greely HT (December 2018). "Is it 'gene therapy'?". Journal of Law and the Biosciences. 5 (3): 786–793. doi:10.1093/jlb/lsy020. PMC 6534757. PMID 31143463.
Asaleh A, Zhou J, Rahmanian N (November 2022). "Letter to the Editor: A Lesson for the Future-How Semantic Ambiguity Led to the Spread of Anti-Scientific Attitudes About COVID-19 mRNA Vaccines". Human Gene Therapy. 33 (21–22): 1213–1216. doi:10.1089/hum.2022.29223.aas (inactive 12 July 2025). PMID 36375123.{{cite journal}}: CS1 maint: DOI inactive as of July 2025 (link)
Williams DA, Orkin SH (April 1986). "Somatic gene therapy. Current status and future prospects". The Journal of Clinical Investigation. 77 (4): 1053–6. doi:10.1172/JCI112403. PMC 424438. PMID 3514670.
Mavilio F, Ferrari G (July 2008). "Genetic modification of somatic stem cells. The progress, problems and prospects of a new therapeutic technology". EMBO Reports. 9 (Suppl 1): S64–69. doi:10.1038/embor.2008.81. PMC 3327547. PMID 18578029.
Mullard A (June 2020). "Gene-editing pipeline takes off". Nature Reviews. Drug Discovery. 19 (6): 367–372. doi:10.1038/d41573-020-00096-y. PMID 32415249. S2CID 218657910.
MacLaren RE, Groppe M, Barnard AR, Cottriall CL, Tolmachova T, Seymour L, Clark KR, During MJ, Cremers FP, Black GC, Lotery AJ, Downes SM, Webster AR, Seabra MC (March 2014). "Retinal gene therapy in patients with choroideremia: initial findings from a phase 1/2 clinical trial". Lancet. 383 (9923): 1129–1137. doi:10.1016/S0140-6736(13)62117-0. PMC 4171740. PMID 24439297.
Dever DP, Bak RO, Reinisch A, Camarena J, Washington G, Nicolas CE, et al. (November 2016). "CRISPR/Cas9 β-globin gene targeting in human haematopoietic stem cells". Nature. 539 (7629): 384–389. Bibcode:2016Natur.539..384D. doi:10.1038/nature20134. PMC 5898607. PMID 27820943.
Gupta RM, Musunuru K (October 2014). "Expanding the genetic editing tool kit: ZFNs, TALENs, and CRISPR-Cas9". The Journal of Clinical Investigation. 124 (10): 4154–61. Bibcode:2014JCliI.124.4154G. doi:10.1172/JCI72992. PMC 4191047. PMID 25271723.
Sanches-da-Silva GN, Medeiros LF, Lima FM (21 August 2019). "The Potential Use of the CRISPR-Cas System for HIV-1 Gene Therapy". International Journal of Genomics. 2019 8458263. doi:10.1155/2019/8458263. PMC 6721108. PMID 31531340.
Bi A, Cui J, Ma YP, Olshevskaya E, Pu M, Dizhoor AM, Pan ZH (April 2006). "Ectopic expression of a microbial-type rhodopsin restores visual responses in mice with photoreceptor degeneration". Neuron. 50 (1): 23–33. doi:10.1016/j.neuron.2006.02.026. PMC 1459045. PMID 16600853.
Song, Xiangrong; Liu, Chao; Wang, Ning; Huang, Hai; He, Siyan; Gong, Changyang; Wei, Yuquan (1 January 2021). "Delivery of CRISPR/Cas systems for cancer gene therapy and immunotherapy". Advanced Drug Delivery Reviews. Delivery of Biomacromolecules for Therapeutic Genome Editing. 168: 158–180. doi:10.1016/j.addr.2020.04.010. ISSN 0169-409X. PMID 32360576. S2CID 218493473.
Nayerossadat N, Maedeh T, Ali PA (2012). "Viral and nonviral delivery systems for gene delivery". Advanced Biomedical Research. 1 (1): 27. doi:10.4103/2277-9175.98152. PMC 3507026. PMID 23210086.
Ginn SL, Amaya AK, Alexander IE, Edelstein M, Abedi MR (May 2018). "Gene therapy clinical trials worldwide to 2017: An update". The Journal of Gene Medicine. 20 (5) e3015. doi:10.1002/jgm.3015. PMID 29575374. S2CID 4966300.
Bulcha JT, Wang Y, Ma H, Tai PW, Gao G (February 2021). "Viral vector platforms within the gene therapy landscape". Signal Transduction and Targeted Therapy. 6 (1) 53. doi:10.1038/s41392-021-00487-6. PMC 7868676. PMID 33558455.
Jensen, Thomas Leth; Gøtzsche, Casper René; Woldbye, David P. D. (2021). "Current and Future Prospects for Gene Therapy for Rare Genetic Diseases Affecting the Brain and Spinal Cord". Frontiers in Molecular Neuroscience. 14 695937. doi:10.3389/fnmol.2021.695937. ISSN 1662-5099. PMC 8527017. PMID 34690692.
Manini A, Abati E, Nuredini A, Corti S, Comi GP (2022). "Adeno-Associated Virus (AAV)-Mediated Gene Therapy for Duchenne Muscular Dystrophy: The Issue of Transgene Persistence". Frontiers in Neurology. 12 814174. doi:10.3389/fneur.2021.814174. PMC 8797140. PMID 35095747.
Sabatino DE, Bushman FD, Chandler RJ, Crystal RG, Davidson BL, Dolmetsch R, et al. (August 2022). "Evaluating the state of the science for adeno-associated virus integration: An integrated perspective". Molecular Therapy. 30 (8): 2646–2663. doi:10.1016/j.ymthe.2022.06.004. PMC 9372310. PMID 35690906.
Campochiaro, P. A.; Avery, R.; Brown, D. M.; Heier, J. S.; Ho, A. C.; Huddleston, S. M.; Jaffe, G. J.; Khanani, A. M.; Pakola, S.; Pieramici, D. J.; Wykoff, C. C.; Van Everen, S. (2024). "Gene therapy for neovascular age-related macular degeneration by subretinal delivery of RGX-314: a phase 1/2a dose-escalation study". Lancet. 403 (10436): 1563–1573. doi:10.1016/S0140-6736(24)00310-6. PMID 38554726.
Khanani, A. M.; Boyer, D. S.; Wykoff, C. C.; Regillo, C. D.; Busbee, B. G.; Pieramici, D.; Danzig, C. J.; Joondeph, B. C.; Major Jr, J. C.; Turpcu, A.; Kiss, S. (2024). "Safety and efficacy of ixoberogene soroparvovec in neovascular age-related macular degeneration in the United States (OPTIC): a prospective, two-year, multicentre phase 1 study". eClinicalMedicine. 67 102394. doi:10.1016/j.eclinm.2023.102394. PMC 10751837. PMID 38152412.
Yin H, Kanasty RL, Eltoukhy AA, Vegas AJ, Dorkin JR, Anderson DG (August 2014). "Non-viral vectors for gene-based therapy". Nature Reviews. Genetics. 15 (8): 541–555. doi:10.1038/nrg3763. PMID 25022906. S2CID 15273455.
Krasilnikova, O.; Yakimova, A.; Ivanov, S.; Atiakshin, D.; Kostin, A.A.; Sosin, D.; Shegay, P.; Kaprin, A.D.; Klabukov, I. (2023). "Gene-Activated Materials in Regenerative Dentistry: Narrative Review of Technology and Study Results". International Journal of Molecular Sciences. 24 (22) 16250. doi:10.3390/ijms242216250. ISSN 1422-0067. PMC 10671237. PMID 38003439.
Foldvari, M.; Chen, D.W.; Nafissi, N.; Calderon, D.; Narsineni, L.; Rafiee, A. (2016). "Non-viral gene therapy: Gains and challenges of non-invasive administration methods". Journal of Controlled Release. 240: 165–190. doi:10.1016/j.jconrel.2015.12.012. ISSN 1873-4995. PMID 26686079.
Bertrand N, Grenier P, Mahmoudi M, Lima EM, Appel EA, Dormont F, et al. (October 2017). "Mechanistic understanding of in vivo protein corona formation on polymeric nanoparticles and impact on pharmacokinetics". Nature Communications. 8 (1) 777. Bibcode:2017NatCo...8..777B. doi:10.1038/s41467-017-00600-w. PMC 5626760. PMID 28974673.
Mennati, Afsaneh; Rostamizadeh, Kobra; Fathi, Mojtaba (1 March 2025). "Dual silencing of integrin αvβ3 receptor and insulin-like growth factor 1 receptor using mPEG-PCL/DDAB hybrid nanoparticle loaded siRNA in breast cancer therapy: An in vitro study on MCF-7 cells". International Journal of Biological Macromolecules. 294 139334. doi:10.1016/j.ijbiomac.2024.139334. ISSN 0141-8130. PMID 39743068.
Goswami R, Subramanian G, Silayeva L, Newkirk I, Doctor D, Chawla K, et al. (24 April 2019). "Gene Therapy Leaves a Vicious Cycle". Frontiers in Oncology. 9 297. doi:10.3389/fonc.2019.00297. PMC 6491712. PMID 31069169.
Wirth T, Parker N, Ylä-Herttuala S (August 2013). "History of gene therapy". Gene. 525 (2): 162–169. doi:10.1016/j.gene.2013.03.137. PMID 23618815.
Singh V, Khan N, Jayandharan GR (May 2022). "Vector engineering, strategies and targets in cancer gene therapy". Cancer Gene Therapy. 29 (5): 402–417. doi:10.1038/s41417-021-00331-7. PMID 33859378. S2CID 233258994.
Anguela XM, High KA (January 2019). "Entering the Modern Era of Gene Therapy". Annual Review of Medicine. 70 (1): 273–288. doi:10.1146/annurev-med-012017-043332. PMID 30477394. S2CID 53750215.
Wang Y, Zhang R, Tang L, Yang L (February 2022). "Nonviral Delivery Systems of mRNA Vaccines for Cancer Gene Therapy". Pharmaceutics. 14 (3): 512. doi:10.3390/pharmaceutics14030512. PMC 8949480. PMID 35335891.
Keam, Susan J. (November 2024). "Afamitresgene Autoleucel: First Approval". Molecular Diagnosis & Therapy. 28 (6): 861–866. doi:10.1007/s40291-024-00749-3. ISSN 1179-2000. PMID 39404764.
Monga I, Qureshi A, Thakur N, Gupta AK, Kumar M (September 2017). "ASPsiRNA: A Resource of ASP-siRNAs Having Therapeutic Potential for Human Genetic Disorders and Algorithm for Prediction of Their Inhibitory Efficacy". G3. 7 (9): 2931–2943. doi:10.1534/g3.117.044024. PMC 5592921. PMID 28696921.
Olowoyeye A, Okwundu CI (November 2020). "Gene therapy for sickle cell disease". The Cochrane Database of Systematic Reviews. 2020 (11) CD007652. doi:10.1002/14651858.CD007652.pub7. PMC 8275984. PMID 33251574.
Scott, Lesley J. (February 2015). "Alipogene tiparvovec: a review of its use in adults with familial lipoprotein lipase deficiency". Drugs. 75 (2): 175–182. doi:10.1007/s40265-014-0339-9. ISSN 1179-1950. PMID 25559420.
Guo, Congting; Ma, Xiaoteng; Gao, Fei; Guo, Yuxuan (9 March 2023). "Off-target effects in CRISPR/Cas9 gene editing". Frontiers in Bioengineering and Biotechnology. 11 1143157. doi:10.3389/fbioe.2023.1143157. ISSN 2296-4185. PMC 10034092. PMID 36970624.
Wu, Shao-Shuai; Li, Qing-Cui; Yin, Chang-Qing; Xue, Wen; Song, Chun-Qing (15 March 2020). "Advances in CRISPR/Cas-based Gene Therapy in Human Genetic Diseases". Theranostics. 10 (10): 4374–4382. doi:10.7150/thno.43360. ISSN 1838-7640. PMC 7150498. PMID 32292501.
Woods NB, Bottero V, Schmidt M, von Kalle C, Verma IM (April 2006). "Gene therapy: therapeutic gene causing lymphoma". Nature. 440 (7088): 1123. Bibcode:2006Natur.440.1123W. doi:10.1038/4401123a. PMID 16641981. S2CID 4372110.
Thrasher AJ, Gaspar HB, Baum C, Modlich U, Schambach A, Candotti F, et al. (September 2006). "Gene therapy: X-SCID transgene leukaemogenicity". Nature. 443 (7109): E5-6, discussion E6-7. Bibcode:2006Natur.443E...5T. doi:10.1038/nature05219. PMID 16988659. S2CID 4301764.
Bak RO, Porteus MH (July 2017). "CRISPR-Mediated Integration of Large Gene Cassettes Using AAV Donor Vectors". Cell Reports. 20 (3): 750–756. doi:10.1016/j.celrep.2017.06.064. PMC 5568673. PMID 28723575.
Raper SE, Chirmule N, Lee FS, Wivel NA, Bagg A, Gao GP, et al. (September 2003). "Fatal systemic inflammatory response syndrome in a ornithine transcarbamylase deficient patient following adenoviral gene transfer". Molecular Genetics and Metabolism. 80 (1–2): 148–58. doi:10.1016/j.ymgme.2003.08.016. PMID 14567964.
Frank KM, Hogarth DK, Miller JL, Mandal S, Mease PJ, Samulski RJ, et al. (July 2009). "Investigation of the cause of death in a gene-therapy trial". The New England Journal of Medicine. 361 (2): 161–9. doi:10.1056/NEJMoa0801066. PMID 19587341.
Kayser B, Mauron A, Miah A (March 2007). "Current anti-doping policy: a critical appraisal". BMC Medical Ethics. 8 2. doi:10.1186/1472-6939-8-2. PMC 1851967. PMID 17394662.
Powell R, Buchanan A (February 2011). "Breaking evolution's chains: the prospect of deliberate genetic modification in humans". The Journal of Medicine and Philosophy. 36 (1): 6–27. doi:10.1093/jmp/jhq057. PMID 21228084.
Baylis F, Robert JS (2004). "The inevitability of genetic enhancement technologies". Bioethics. 18 (1): 1–26. doi:10.1111/j.1467-8519.2004.00376.x. PMID 15168695.
Allhoff F (March 2005). "Germ-line genetic enhancement and Rawlsian primary goods" (PDF). Kennedy Institute of Ethics Journal. 15 (1): 39–56. CiteSeerX 10.1.1.566.171. doi:10.1353/ken.2005.0007. PMID 15881795. S2CID 14432440. Archived (PDF) from the original on 30 January 2012.
"Ethical issues related to prenatal genetic testing. The Council on Ethical and Judicial Affairs, American Medical Association". Archives of Family Medicine. 3 (7): 633–642. July 1994. doi:10.1001/archfami.3.7.633. PMID 7921302.
Smith KR, Chan S, Harris J (October 2012). "Human germline genetic modification: scientific and bioethical perspectives". Archives of Medical Research. 43 (7): 491–513. doi:10.1016/j.arcmed.2012.09.003. PMID 23072719.
Baltimore D, Berg P, Botchan M, Carroll D, Charo RA, Church G, et al. (April 2015). "Biotechnology. A prudent path forward for genomic engineering and germline gene modification". Science. 348 (6230): 36–38. Bibcode:2015Sci...348...36B. doi:10.1126/science.aab1028. PMC 4394183. PMID 25791083.
Lanphier E, Urnov F, Haecker SE, Werner M, Smolenski J (March 2015). "Don't edit the human germ line". Nature. 519 (7544): 410–411. Bibcode:2015Natur.519..410L. doi:10.1038/519410a. PMID 25810189.
Liang P, Xu Y, Zhang X, Ding C, Huang R, Zhang Z, et al. (May 2015). "CRISPR/Cas9-mediated gene editing in human tripronuclear zygotes". Protein & Cell. 6 (5): 363–372. doi:10.1007/s13238-015-0153-5. PMC 4417674. PMID 25894090.
Friedmann T, Roblin R (March 1972). "Gene therapy for human genetic disease?". Science. 175 (4025): 949–955. Bibcode:1972Sci...175..949F. doi:10.1126/science.175.4025.949. PMID 5061866. S2CID 19952096.
Cepko CL, Roberts BE, Mulligan RC (July 1984). "Construction and applications of a highly transmissible murine retrovirus shuttle vector. This Vector is used for entering a cell in the humans cell body". Cell. 37 (3): 1053–1062. doi:10.1016/0092-8674(84)90440-9. PMID 6331674. S2CID 34544709.
Blaese RM, Culver KW, Miller AD, Carter CS, Fleisher T, Clerici M, et al. (October 1995). "T lymphocyte-directed gene therapy for ADA- SCID: initial trial results after 4 years". Science. 270 (5235): 475–480. Bibcode:1995Sci...270..475B. doi:10.1126/science.270.5235.475. PMID 7570001. S2CID 46339645.
Trojan J, Johnson TR, Rudin SD, Ilan J, Tykocinski ML, Ilan J (January 1993). "Treatment and prevention of rat glioblastoma by immunogenic C6 cells expressing antisense insulin-like growth factor I RNA". Science. 259 (5091): 94–97. Bibcode:1993Sci...259...94T. doi:10.1126/science.8418502. PMID 8418502.
Trojan J, Pan YX, Wei MX, Ly A, Shevelev A, Bierwagen M, et al. (2012). "Methodology for Anti-Gene Anti-IGF-I Therapy of Malignant Tumours". Chemotherapy Research and Practice. 2012: 1–12. doi:10.1155/2012/721873. PMC 3287029. PMID 22400112.
Abbott A (April 1992). "Gene therapy. Italians first to use stem cells". Nature. 356 (6369): 465. Bibcode:1992Natur.356..465A. doi:10.1038/356465a0. PMID 1560817. S2CID 4319842.
Cavazzana-Calvo M, Thrasher A, Mavilio F (February 2004). "The future of gene therapy". Nature. 427 (6977): 779–781. Bibcode:2004Natur.427..779C. doi:10.1038/427779a. hdl:11380/307566. PMID 14985734. S2CID 4421364.
Naldini, Luigi; Blömer, Ulrike; Gallay, Philippe; Ory, Daniel; Mulligan, Richard; Gage, Fred H.; Verma, Inder M.; Trono, Didier (12 April 1996). "In Vivo Gene Delivery and Stable Transduction of Nondividing Cells by a Lentiviral Vector". Science. 272 (5259): 263–267. Bibcode:1996Sci...272..263N. doi:10.1126/science.272.5259.263. ISSN 0036-8075. PMID 8602510. S2CID 18997464.
Ott MG, Schmidt M, Schwarzwaelder K, Stein S, Siler U, Koehl U, et al. (April 2006). "Correction of X-linked chronic granulomatous disease by gene therapy, augmented by insertional activation of MDS1-EVI1, PRDM16 or SETBP1". Nature Medicine. 12 (4): 401–409. doi:10.1038/nm1393. PMID 16582916. S2CID 7601162.
Brown BD, Venneri MA, Zingale A, Sergi Sergi L, Naldini L (May 2006). "Endogenous microRNA regulation suppresses transgene expression in hematopoietic lineages and enables stable gene transfer". Nature Medicine. 12 (5): 585–591. doi:10.1038/nm1398. PMID 16633348. S2CID 11114427.
Morgan RA, Dudley ME, Wunderlich JR, Hughes MS, Yang JC, Sherry RM, et al. (October 2006). "Cancer regression in patients after transfer of genetically engineered lymphocytes". Science. 314 (5796): 126–129. Bibcode:2006Sci...314..126M. doi:10.1126/science.1129003. PMC 2267026. PMID 16946036.
Levine BL, Humeau LM, Boyer J, MacGregor RR, Rebello T, Lu X, et al. (November 2006). "Gene transfer in humans using a conditionally replicating lentiviral vector". Proceedings of the National Academy of Sciences of the United States of America. 103 (46): 17372–17377. Bibcode:2006PNAS..10317372L. doi:10.1073/pnas.0608138103. PMC 1635018. PMID 17090675.
Cartier N, Hacein-Bey-Abina S, Bartholomae CC, Veres G, Schmidt M, Kutschera I, et al. (November 2009). "Hematopoietic stem cell gene therapy with a lentiviral vector in X-linked adrenoleukodystrophy" (PDF). Science. 326 (5954): 818–823. Bibcode:2009Sci...326..818C. doi:10.1126/science.1171242. PMID 19892975. S2CID 27783.
Komáromy AM, Alexander JJ, Rowlan JS, Garcia MM, Chiodo VA, Kaya A, et al. (July 2010). "Gene therapy rescues cone function in congenital achromatopsia". Human Molecular Genetics. 19 (13): 2581–2593. doi:10.1093/hmg/ddq136. PMC 2883338. PMID 20378608.
Cavazzana-Calvo M, Payen E, Negre O, Wang G, Hehir K, Fusil F, et al. (September 2010). "Transfusion independence and HMGA2 activation after gene therapy of human β-thalassaemia". Nature. 467 (7313): 318–322. Bibcode:2010Natur.467..318C. doi:10.1038/nature09328. PMC 3355472. PMID 20844535.
Galanello R, Origa R (May 2010). "Beta-thalassemia". Orphanet Journal of Rare Diseases. 5 11. doi:10.1186/1750-1172-5-11. PMC 2893117. PMID 20492708.
Yang ZJ, Zhang YR, Chen B, Zhang SL, Jia EZ, Wang LS, et al. (July 2009). "Phase I clinical trial on intracoronary administration of Ad-hHGF treating severe coronary artery disease". Molecular Biology Reports. 36 (6): 1323–1329. doi:10.1007/s11033-008-9315-3. PMID 18649012. S2CID 23419866.
Hahn W, Pyun WB, Kim DS, Yoo WS, Lee SD, Won JH, et al. (October 2011). "Enhanced cardioprotective effects by coexpression of two isoforms of hepatocyte growth factor from naked plasmid DNA in a rat ischemic heart disease model". The Journal of Gene Medicine. 13 (10): 549–555. doi:10.1002/jgm.1603. PMID 21898720. S2CID 26812780.
Deev RV, Bozo IY, Mzhavanadze ND, Voronov DA, Gavrilenko AV, Chervyakov YV, et al. (September 2015). "pCMV-vegf165 Intramuscular Gene Transfer is an Effective Method of Treatment for Patients With Chronic Lower Limb Ischemia". Journal of Cardiovascular Pharmacology and Therapeutics. 20 (5): 473–482. doi:10.1177/1074248415574336. PMID 25770117. S2CID 13443907.
Morrison C (3 March 2015). "$1-million price tag set for Glybera gene therapy". TradeSecrets. 33 (3): 217–218. doi:10.1038/nbt0315-217. PMID 25748892. S2CID 205266596.
Fernàndez-Ruiz I (March 2016). "Gene therapy: No improvement in outcomes with gene therapy for heart failure". Nature Reviews. Cardiology. 13 (3): 122–123. doi:10.1038/nrcardio.2016.14. PMID 26843287. S2CID 205472001.
Biffi A, Montini E, Lorioli L, Cesani M, Fumagalli F, Plati T, et al. (August 2013). "Lentiviral hematopoietic stem cell gene therapy benefits metachromatic leukodystrophy". Science. 341 (6148) 1233158. doi:10.1126/science.1233158. PMID 23845948. S2CID 206546808.
Aiuti A, Biasco L, Scaramuzza S, Ferrua F, Cicalese MP, Baricordi C, et al. (August 2013). "Lentiviral hematopoietic stem cell gene therapy in patients with Wiskott–Aldrich syndrome". Science. 341 (6148) 1233151. doi:10.1126/science.1233151. PMC 4375961. PMID 23845947.
Malech HL, Ochs HD (April 2015). "An emerging era of clinical benefit from gene therapy". JAMA. 313 (15): 1522–1523. doi:10.1001/jama.2015.2055. PMID 25898049.
Tebas P, Stein D, Tang WW, Frank I, Wang SQ, Lee G, et al. (March 2014). "Gene editing of CCR5 in autologous CD4 T cells of persons infected with HIV". The New England Journal of Medicine. 370 (10): 901–910. doi:10.1056/NEJMoa1300662. PMC 4084652. PMID 24597865.
Gardner MR, Kattenhorn LM, Kondur HR, von Schaewen M, Dorfman T, Chiang JJ, et al. (March 2015). "AAV-expressed eCD4-Ig provides durable protection from multiple SHIV challenges". Nature. 519 (7541): 87–91. Bibcode:2015Natur.519...87G. doi:10.1038/nature14264. PMC 4352131. PMID 25707797.
Cyranoski D (July 2016). "Chinese scientists to pioneer first human CRISPR trial". Nature. 535 (7613): 476–477. Bibcode:2016Natur.535..476C. doi:10.1038/nature.2016.20302. PMID 27466105.
Lee TW, Southern KW, Perry LA, Penny-Dimri JC, Aslam AA (June 2016). Southern KW (ed.). "Topical cystic fibrosis transmembrane conductance regulator gene replacement for cystic fibrosis-related lung disease". The Cochrane Database of Systematic Reviews. 2016 (6) CD005599. doi:10.1002/14651858.CD005599.pub5. PMC 8682957. PMID 27314455.
Rangarajan S, Walsh L, Lester W, Perry D, Madan B, Laffan M, et al. (December 2017). "AAV5-Factor VIII Gene Transfer in Severe Hemophilia A". The New England Journal of Medicine. 377 (26): 2519–2530. doi:10.1056/nejmoa1708483. hdl:10044/1/57163. PMID 29224506.
van den Berg HM (December 2017). "A Cure for Hemophilia within Reach". The New England Journal of Medicine. 377 (26): 2592–2593. doi:10.1056/nejme1713888. PMID 29224412.
Ledford H (March 2020). "CRISPR treatment inserted directly into the body for first time". Nature. 579 (7798): 185. Bibcode:2020Natur.579..185L. doi:10.1038/d41586-020-00655-8. PMID 32157225.
"High-dose AAV gene therapy deaths". Nature Biotechnology. 38 (8): 910. 1 August 2020. doi:10.1038/s41587-020-0642-9. ISSN 1546-1696. PMID 32760031. S2CID 220981004.
Kohn DB, Booth C, Shaw KL, Xu-Bayford J, Garabedian E, Trevisan V, et al. (May 2021). "Autologous Ex Vivo Lentiviral Gene Therapy for Adenosine Deaminase Deficiency". The New England Journal of Medicine. 384 (21): 2002–2013. doi:10.1056/NEJMoa2027675. PMC 8240285. PMID 33974366.
Mamcarz E, Zhou S, Lockey T, Abdelsamed H, Cross SJ, Kang G, et al. (April 2019). "Lentiviral Gene Therapy Combined with Low-Dose Busulfan in Infants with SCID-X1". The New England Journal of Medicine. 380 (16): 1525–1534. doi:10.1056/NEJMoa1815408. PMC 6636624. PMID 30995372.
Gillmore JD, Gane E, Taubel J, Kao J, Fontana M, Maitland ML, et al. (August 2021). "CRISPR-Cas9 In Vivo Gene Editing for Transthyretin Amyloidosis". The New England Journal of Medicine. 385 (6): 493–502. doi:10.1056/NEJMoa2107454. PMID 34215024. S2CID 235722446.
"The science news that shaped 2021: Nature's picks". Nature. December 2021. doi:10.1038/d41586-021-03734-6. PMID 34907370. S2CID 245144020.
Pearson TS, Gupta N, San Sebastian W, Imamura-Ching J, Viehoever A, Grijalvo-Perez A, et al. (July 2021). "Gene therapy for aromatic L-amino acid decarboxylase deficiency by MR-guided direct delivery of AAV2-AADC to midbrain dopaminergic neurons". Nature Communications. 12 (1) 4251. Bibcode:2021NatCo..12.4251P. doi:10.1038/s41467-021-24524-8. PMC 8275582. PMID 34253733.
Flotte TR, Cataltepe O, Puri A, Batista AR, Moser R, McKenna-Yasek D, et al. (February 2022). "AAV gene therapy for Tay-Sachs disease". Nature Medicine. 28 (2): 251–259. doi:10.1038/s41591-021-01664-4. PMC 10786171. PMID 35145305. S2CID 246748772.
Chowdary P, Shapiro S, Makris M, Evans G, Boyce S, Talks K, et al. (July 2022). "Phase 1-2 Trial of AAVS3 Gene Therapy in Patients with Hemophilia B". The New England Journal of Medicine. 387 (3): 237–247. doi:10.1056/NEJMoa2119913. PMID 35857660. S2CID 250697905.
Huang, Jinbo; Xue, Shuai; Buchmann, Peter; Teixeira, Ana Palma; Fussenegger, Martin (16 August 2023). "An electrogenetic interface to program mammalian gene expression by direct current". Nature Metabolism. 5 (8): 1395–1407. doi:10.1038/s42255-023-00850-7. PMC 10447240. PMID 37524785.

ncbi.nlm.nih.gov

Gorell E, Nguyen N, Lane A, Siprashvili Z (April 2014). "Gene therapy for skin diseases". Cold Spring Harbor Perspectives in Medicine. 4 (4) a015149. doi:10.1101/cshperspect.a015149. PMC 3968787. PMID 24692191.
Maguire AM, Simonelli F, Pierce EA, Pugh EN, Mingozzi F, Bennicelli J, et al. (May 2008). "Safety and efficacy of gene transfer for Leber's congenital amaurosis". The New England Journal of Medicine. 358 (21): 2240–2248. doi:10.1056/NEJMoa0802315. PMC 2829748. PMID 18441370.
Simonelli F, Maguire AM, Testa F, Pierce EA, Mingozzi F, Bennicelli JL, et al. (March 2010). "Gene therapy for Leber's congenital amaurosis is safe and effective through 1.5 years after vector administration". Molecular Therapy. 18 (3): 643–650. doi:10.1038/mt.2009.277. PMC 2839440. PMID 19953081.
Cideciyan AV, Hauswirth WW, Aleman TS, Kaushal S, Schwartz SB, Boye SL, Windsor EA, Conlon TJ, Sumaroka A, Roman AJ, Byrne BJ, Jacobson SG (August 2009). "Vision 1 year after gene therapy for Leber's congenital amaurosis". The New England Journal of Medicine. 361 (7): 725–727. doi:10.1056/NEJMc0903652. PMC 2847775. PMID 19675341.
Cartier N, Aubourg P (July 2010). "Hematopoietic stem cell transplantation and hematopoietic stem cell gene therapy in X-linked adrenoleukodystrophy". Brain Pathology. 20 (4): 857–862. doi:10.1111/j.1750-3639.2010.00394.x. PMC 8094635. PMID 20626747. S2CID 24182017.
Pearson S, Jia H, Kandachi K (January 2004). "China approves first gene therapy". Nature Biotechnology. 22 (1): 3–4. doi:10.1038/nbt0104-3. PMC 7097065. PMID 14704685.
Gothelf A, Gehl J (November 2012). "What you always needed to know about electroporation based DNA vaccines". Human Vaccines & Immunotherapeutics. 8 (11): 1694–1702. doi:10.4161/hv.22062. PMC 3601144. PMID 23111168.
Stone D, Niyonzima N, Jerome KR (September 2016). "Genome editing and the next generation of antiviral therapy". Human Genetics. 135 (9): 1071–82. doi:10.1007/s00439-016-1686-2. PMC 5002242. PMID 27272125.
Cross D, Burmester JK (September 2006). "Gene therapy for cancer treatment: past, present and future". Clinical Medicine & Research. 4 (3): 218–27. doi:10.3121/cmr.4.3.218. PMC 1570487. PMID 16988102.
Maeder ML, Gersbach CA (March 2016). "Genome-editing Technologies for Gene and Cell Therapy". Molecular Therapy. 24 (3): 430–46. doi:10.1038/mt.2016.10. PMC 4786923. PMID 26755333.
Sherkow JS, Zettler PJ, Greely HT (December 2018). "Is it 'gene therapy'?". Journal of Law and the Biosciences. 5 (3): 786–793. doi:10.1093/jlb/lsy020. PMC 6534757. PMID 31143463.
Williams DA, Orkin SH (April 1986). "Somatic gene therapy. Current status and future prospects". The Journal of Clinical Investigation. 77 (4): 1053–6. doi:10.1172/JCI112403. PMC 424438. PMID 3514670.
Mavilio F, Ferrari G (July 2008). "Genetic modification of somatic stem cells. The progress, problems and prospects of a new therapeutic technology". EMBO Reports. 9 (Suppl 1): S64–69. doi:10.1038/embor.2008.81. PMC 3327547. PMID 18578029.
MacLaren RE, Groppe M, Barnard AR, Cottriall CL, Tolmachova T, Seymour L, Clark KR, During MJ, Cremers FP, Black GC, Lotery AJ, Downes SM, Webster AR, Seabra MC (March 2014). "Retinal gene therapy in patients with choroideremia: initial findings from a phase 1/2 clinical trial". Lancet. 383 (9923): 1129–1137. doi:10.1016/S0140-6736(13)62117-0. PMC 4171740. PMID 24439297.
Dever DP, Bak RO, Reinisch A, Camarena J, Washington G, Nicolas CE, et al. (November 2016). "CRISPR/Cas9 β-globin gene targeting in human haematopoietic stem cells". Nature. 539 (7629): 384–389. Bibcode:2016Natur.539..384D. doi:10.1038/nature20134. PMC 5898607. PMID 27820943.
Gupta RM, Musunuru K (October 2014). "Expanding the genetic editing tool kit: ZFNs, TALENs, and CRISPR-Cas9". The Journal of Clinical Investigation. 124 (10): 4154–61. Bibcode:2014JCliI.124.4154G. doi:10.1172/JCI72992. PMC 4191047. PMID 25271723.
Sanches-da-Silva GN, Medeiros LF, Lima FM (21 August 2019). "The Potential Use of the CRISPR-Cas System for HIV-1 Gene Therapy". International Journal of Genomics. 2019 8458263. doi:10.1155/2019/8458263. PMC 6721108. PMID 31531340.
Bi A, Cui J, Ma YP, Olshevskaya E, Pu M, Dizhoor AM, Pan ZH (April 2006). "Ectopic expression of a microbial-type rhodopsin restores visual responses in mice with photoreceptor degeneration". Neuron. 50 (1): 23–33. doi:10.1016/j.neuron.2006.02.026. PMC 1459045. PMID 16600853.
Nayerossadat N, Maedeh T, Ali PA (2012). "Viral and nonviral delivery systems for gene delivery". Advanced Biomedical Research. 1 (1): 27. doi:10.4103/2277-9175.98152. PMC 3507026. PMID 23210086.
Bulcha JT, Wang Y, Ma H, Tai PW, Gao G (February 2021). "Viral vector platforms within the gene therapy landscape". Signal Transduction and Targeted Therapy. 6 (1) 53. doi:10.1038/s41392-021-00487-6. PMC 7868676. PMID 33558455.
Jensen, Thomas Leth; Gøtzsche, Casper René; Woldbye, David P. D. (2021). "Current and Future Prospects for Gene Therapy for Rare Genetic Diseases Affecting the Brain and Spinal Cord". Frontiers in Molecular Neuroscience. 14 695937. doi:10.3389/fnmol.2021.695937. ISSN 1662-5099. PMC 8527017. PMID 34690692.
Manini A, Abati E, Nuredini A, Corti S, Comi GP (2022). "Adeno-Associated Virus (AAV)-Mediated Gene Therapy for Duchenne Muscular Dystrophy: The Issue of Transgene Persistence". Frontiers in Neurology. 12 814174. doi:10.3389/fneur.2021.814174. PMC 8797140. PMID 35095747.
Sabatino DE, Bushman FD, Chandler RJ, Crystal RG, Davidson BL, Dolmetsch R, et al. (August 2022). "Evaluating the state of the science for adeno-associated virus integration: An integrated perspective". Molecular Therapy. 30 (8): 2646–2663. doi:10.1016/j.ymthe.2022.06.004. PMC 9372310. PMID 35690906.
Khanani, A. M.; Boyer, D. S.; Wykoff, C. C.; Regillo, C. D.; Busbee, B. G.; Pieramici, D.; Danzig, C. J.; Joondeph, B. C.; Major Jr, J. C.; Turpcu, A.; Kiss, S. (2024). "Safety and efficacy of ixoberogene soroparvovec in neovascular age-related macular degeneration in the United States (OPTIC): a prospective, two-year, multicentre phase 1 study". eClinicalMedicine. 67 102394. doi:10.1016/j.eclinm.2023.102394. PMC 10751837. PMID 38152412.
Krasilnikova, O.; Yakimova, A.; Ivanov, S.; Atiakshin, D.; Kostin, A.A.; Sosin, D.; Shegay, P.; Kaprin, A.D.; Klabukov, I. (2023). "Gene-Activated Materials in Regenerative Dentistry: Narrative Review of Technology and Study Results". International Journal of Molecular Sciences. 24 (22) 16250. doi:10.3390/ijms242216250. ISSN 1422-0067. PMC 10671237. PMID 38003439.
Bertrand N, Grenier P, Mahmoudi M, Lima EM, Appel EA, Dormont F, et al. (October 2017). "Mechanistic understanding of in vivo protein corona formation on polymeric nanoparticles and impact on pharmacokinetics". Nature Communications. 8 (1) 777. Bibcode:2017NatCo...8..777B. doi:10.1038/s41467-017-00600-w. PMC 5626760. PMID 28974673.
Goswami R, Subramanian G, Silayeva L, Newkirk I, Doctor D, Chawla K, et al. (24 April 2019). "Gene Therapy Leaves a Vicious Cycle". Frontiers in Oncology. 9 297. doi:10.3389/fonc.2019.00297. PMC 6491712. PMID 31069169.
Wang Y, Zhang R, Tang L, Yang L (February 2022). "Nonviral Delivery Systems of mRNA Vaccines for Cancer Gene Therapy". Pharmaceutics. 14 (3): 512. doi:10.3390/pharmaceutics14030512. PMC 8949480. PMID 35335891.
Monga I, Qureshi A, Thakur N, Gupta AK, Kumar M (September 2017). "ASPsiRNA: A Resource of ASP-siRNAs Having Therapeutic Potential for Human Genetic Disorders and Algorithm for Prediction of Their Inhibitory Efficacy". G3. 7 (9): 2931–2943. doi:10.1534/g3.117.044024. PMC 5592921. PMID 28696921.
Olowoyeye A, Okwundu CI (November 2020). "Gene therapy for sickle cell disease". The Cochrane Database of Systematic Reviews. 2020 (11) CD007652. doi:10.1002/14651858.CD007652.pub7. PMC 8275984. PMID 33251574.
Guo, Congting; Ma, Xiaoteng; Gao, Fei; Guo, Yuxuan (9 March 2023). "Off-target effects in CRISPR/Cas9 gene editing". Frontiers in Bioengineering and Biotechnology. 11 1143157. doi:10.3389/fbioe.2023.1143157. ISSN 2296-4185. PMC 10034092. PMID 36970624.
Wu, Shao-Shuai; Li, Qing-Cui; Yin, Chang-Qing; Xue, Wen; Song, Chun-Qing (15 March 2020). "Advances in CRISPR/Cas-based Gene Therapy in Human Genetic Diseases". Theranostics. 10 (10): 4374–4382. doi:10.7150/thno.43360. ISSN 1838-7640. PMC 7150498. PMID 32292501.
Bak RO, Porteus MH (July 2017). "CRISPR-Mediated Integration of Large Gene Cassettes Using AAV Donor Vectors". Cell Reports. 20 (3): 750–756. doi:10.1016/j.celrep.2017.06.064. PMC 5568673. PMID 28723575.
"Home - NIH Genetic Testing Registry (GTR) - NCBI". www.ncbi.nlm.nih.gov. Retrieved 2 May 2022.
Kayser B, Mauron A, Miah A (March 2007). "Current anti-doping policy: a critical appraisal". BMC Medical Ethics. 8 2. doi:10.1186/1472-6939-8-2. PMC 1851967. PMID 17394662.
Baltimore D, Berg P, Botchan M, Carroll D, Charo RA, Church G, et al. (April 2015). "Biotechnology. A prudent path forward for genomic engineering and germline gene modification". Science. 348 (6230): 36–38. Bibcode:2015Sci...348...36B. doi:10.1126/science.aab1028. PMC 4394183. PMID 25791083.
Liang P, Xu Y, Zhang X, Ding C, Huang R, Zhang Z, et al. (May 2015). "CRISPR/Cas9-mediated gene editing in human tripronuclear zygotes". Protein & Cell. 6 (5): 363–372. doi:10.1007/s13238-015-0153-5. PMC 4417674. PMID 25894090.
Trojan J, Pan YX, Wei MX, Ly A, Shevelev A, Bierwagen M, et al. (2012). "Methodology for Anti-Gene Anti-IGF-I Therapy of Malignant Tumours". Chemotherapy Research and Practice. 2012: 1–12. doi:10.1155/2012/721873. PMC 3287029. PMID 22400112.
Morgan RA, Dudley ME, Wunderlich JR, Hughes MS, Yang JC, Sherry RM, et al. (October 2006). "Cancer regression in patients after transfer of genetically engineered lymphocytes". Science. 314 (5796): 126–129. Bibcode:2006Sci...314..126M. doi:10.1126/science.1129003. PMC 2267026. PMID 16946036.
Levine BL, Humeau LM, Boyer J, MacGregor RR, Rebello T, Lu X, et al. (November 2006). "Gene transfer in humans using a conditionally replicating lentiviral vector". Proceedings of the National Academy of Sciences of the United States of America. 103 (46): 17372–17377. Bibcode:2006PNAS..10317372L. doi:10.1073/pnas.0608138103. PMC 1635018. PMID 17090675.
Komáromy AM, Alexander JJ, Rowlan JS, Garcia MM, Chiodo VA, Kaya A, et al. (July 2010). "Gene therapy rescues cone function in congenital achromatopsia". Human Molecular Genetics. 19 (13): 2581–2593. doi:10.1093/hmg/ddq136. PMC 2883338. PMID 20378608.
Cavazzana-Calvo M, Payen E, Negre O, Wang G, Hehir K, Fusil F, et al. (September 2010). "Transfusion independence and HMGA2 activation after gene therapy of human β-thalassaemia". Nature. 467 (7313): 318–322. Bibcode:2010Natur.467..318C. doi:10.1038/nature09328. PMC 3355472. PMID 20844535.
Galanello R, Origa R (May 2010). "Beta-thalassemia". Orphanet Journal of Rare Diseases. 5 11. doi:10.1186/1750-1172-5-11. PMC 2893117. PMID 20492708.
Aiuti A, Biasco L, Scaramuzza S, Ferrua F, Cicalese MP, Baricordi C, et al. (August 2013). "Lentiviral hematopoietic stem cell gene therapy in patients with Wiskott–Aldrich syndrome". Science. 341 (6148) 1233151. doi:10.1126/science.1233151. PMC 4375961. PMID 23845947.
Tebas P, Stein D, Tang WW, Frank I, Wang SQ, Lee G, et al. (March 2014). "Gene editing of CCR5 in autologous CD4 T cells of persons infected with HIV". The New England Journal of Medicine. 370 (10): 901–910. doi:10.1056/NEJMoa1300662. PMC 4084652. PMID 24597865.
Gardner MR, Kattenhorn LM, Kondur HR, von Schaewen M, Dorfman T, Chiang JJ, et al. (March 2015). "AAV-expressed eCD4-Ig provides durable protection from multiple SHIV challenges". Nature. 519 (7541): 87–91. Bibcode:2015Natur.519...87G. doi:10.1038/nature14264. PMC 4352131. PMID 25707797.
Lee TW, Southern KW, Perry LA, Penny-Dimri JC, Aslam AA (June 2016). Southern KW (ed.). "Topical cystic fibrosis transmembrane conductance regulator gene replacement for cystic fibrosis-related lung disease". The Cochrane Database of Systematic Reviews. 2016 (6) CD005599. doi:10.1002/14651858.CD005599.pub5. PMC 8682957. PMID 27314455.
Kohn DB, Booth C, Shaw KL, Xu-Bayford J, Garabedian E, Trevisan V, et al. (May 2021). "Autologous Ex Vivo Lentiviral Gene Therapy for Adenosine Deaminase Deficiency". The New England Journal of Medicine. 384 (21): 2002–2013. doi:10.1056/NEJMoa2027675. PMC 8240285. PMID 33974366.
Mamcarz E, Zhou S, Lockey T, Abdelsamed H, Cross SJ, Kang G, et al. (April 2019). "Lentiviral Gene Therapy Combined with Low-Dose Busulfan in Infants with SCID-X1". The New England Journal of Medicine. 380 (16): 1525–1534. doi:10.1056/NEJMoa1815408. PMC 6636624. PMID 30995372.
Pearson TS, Gupta N, San Sebastian W, Imamura-Ching J, Viehoever A, Grijalvo-Perez A, et al. (July 2021). "Gene therapy for aromatic L-amino acid decarboxylase deficiency by MR-guided direct delivery of AAV2-AADC to midbrain dopaminergic neurons". Nature Communications. 12 (1) 4251. Bibcode:2021NatCo..12.4251P. doi:10.1038/s41467-021-24524-8. PMC 8275582. PMID 34253733.
Flotte TR, Cataltepe O, Puri A, Batista AR, Moser R, McKenna-Yasek D, et al. (February 2022). "AAV gene therapy for Tay-Sachs disease". Nature Medicine. 28 (2): 251–259. doi:10.1038/s41591-021-01664-4. PMC 10786171. PMID 35145305. S2CID 246748772.
Huang, Jinbo; Xue, Shuai; Buchmann, Peter; Teixeira, Ana Palma; Fussenegger, Martin (16 August 2023). "An electrogenetic interface to program mammalian gene expression by direct current". Nature Metabolism. 5 (8): 1395–1407. doi:10.1038/s42255-023-00850-7. PMC 10447240. PMID 37524785.

nih.gov

"Gene therapy restores immune function in children with rare immunodeficiency". National Institutes of Health (NIH). 11 May 2021. Retrieved 19 July 2021.
Rohr K (17 April 2019). "Gene therapy restores immunity in infants with rare immunodeficiency disease". National Institutes of Health (NIH). Retrieved 4 June 2020.

osp.od.nih.gov

"NIH Guidelines for Research Involving Recombinant or Synthetic Nucleic Acid Molecules". U.S. National Institutes of Health. April 2016. Archived from the original on 28 May 2016.

oar.nih.gov

"Letters from the Director: Remembering Timothy Ray Brown: A Champion for HIV Cure Research | NIH Office of AIDS Research". www.oar.nih.gov. Retrieved 26 June 2022.

npr.org (Global: 92^nd place; English: 72^nd place)

Harris, Richard (8 November 2017). "Genetically Altered Skin Saves A Boy Dying Of A Rare Disease". NPR.org.
Stein R (24 May 2019). "At $2.1 Million, New Gene Therapy Is The Most Expensive Drug Ever". NPR. Retrieved 24 May 2019.
Stein, Rob (29 July 2019). "In A 1st, Doctors In U.S. Use CRISPR Tool To Treat Patient With Genetic Disorder". NPR. Retrieved 25 September 2024.

nymag.com (Global: 346^th place; English: 229^th place)

Rosenberg T (27 May 2011). "The Man Who Was Cured of HIV and What It Means for a Cure for AIDS". New York. Retrieved 2 January 2022.

nypost.com (Global: 208^th place; English: 156^th place)

"Scientists OK genetically engineering babies". New York Post. Reuters. 14 February 2017. Retrieved 17 February 2017.

nytimes.com (Global: 7^th place; English: 7^th place)

Zimmer C (16 September 2013). "DNA Double Take". The New York Times. Archived from the original on 2 January 2022.
Wade N (19 March 2015). "Scientists Seek Ban on Method of Editing the Human Genome". The New York Times. Archived from the original on 2 January 2022. Retrieved 20 March 2015.
Pollack A (3 March 2015). "A Powerful New Way to Edit DNA". The New York Times. Archived from the original on 2 January 2022. Retrieved 20 March 2015.
Kolata G (23 April 2015). "Chinese Scientists Edit Genes of Human Embryos, Raising Concerns". The New York Times. Archived from the original on 2 January 2022. Retrieved 24 April 2015.
Harmon A (14 February 2017). "Human Gene Editing Receives Science Panel's Support". The New York Times. ISSN 0362-4331. Archived from the original on 2 January 2022. Retrieved 17 February 2017.
Blakeslee S (18 May 1993). "Treatment for 'Bubble Boy Disease'". The New York Times. ISSN 0362-4331. Retrieved 9 February 2018.
Stolberg SG (22 January 2000). "Gene Therapy Ordered Halted At University". The New York Times. Retrieved 10 November 2010.
Pollack A (20 July 2012). "European Agency Recommends Approval of a Gene Therapy". The New York Times. Retrieved 2 January 2022.
Zimmer C (9 March 2015). "Protection Without a Vaccine". The New York Times. Archived from the original on 2 January 2022. Retrieved 30 March 2015.
Wade N (3 December 2015). "Scientists Place Moratorium on Edits to Human Genome That Could Be Inherited". The New York Times. Archived from the original on 2 January 2022. Retrieved 3 December 2015.

ornl.gov (Global: 5,534^th place; English: 4,538^th place)

"Gene Therapy". Oak Ridge National Laboratory. 20 December 2012. Archived from the original on 20 December 2012. Retrieved 2 January 2022.

politifact.com (Global: 3,208^th place; English: 1,803^rd place)

"Joe Rogan falsely says mRNA vaccines are 'gene therapy'". PolitiFact. Retrieved 19 February 2023.

popularmechanics.com (Global: 1,808^th place; English: 1,159^th place)

Bennett J (15 November 2016). "Chinese Scientists Become First to Use CRISPR Gene-Editing on Humans". Popular Mechanics. Retrieved 16 November 2016.

psu.edu (Global: 207^th place; English: 136^th place)

citeseerx.ist.psu.edu

Allhoff F (March 2005). "Germ-line genetic enhancement and Rawlsian primary goods" (PDF). Kennedy Institute of Ethics Journal. 15 (1): 39–56. CiteSeerX 10.1.1.566.171. doi:10.1353/ken.2005.0007. PMID 15881795. S2CID 14432440. Archived (PDF) from the original on 30 January 2012.

ptcbio.com (Global: low place; English: low place)

ir.ptcbio.com

"PTC Therapeutics Receives Positive CHMP Opinion for Upstaza for the Treatment of AADC Deficiency". PTC Therapeutics (Press release). 20 May 2022. Archived from the original on 23 May 2022. Retrieved 22 May 2022.
"PTC Therapeutics Announces FDA Approval of AADC Deficiency Gene Therapy". PTC Therapeutics, Inc. 13 November 2024. Archived from the original on 15 November 2024. Retrieved 21 November 2024.

reuters.com (Global: 49^th place; English: 47^th place)

"Fact Check-mRNA vaccines are distinct from gene therapy, which alters recipient's genes". Reuters. 10 August 2021. Retrieved 19 February 2023.
Burger L, Hirschler B (26 November 2014). "First gene therapy drug sets million-euro price record". Reuters. Retrieved 30 March 2015.
Hirscheler B (1 April 2016). "Europe gives green light to first gene therapy for children". Reuters. Retrieved 13 April 2016.

science.org (Global: 1,160^th place; English: 737^th place)

Naldini, Luigi; Blömer, Ulrike; Gallay, Philippe; Ory, Daniel; Mulligan, Richard; Gage, Fred H.; Verma, Inder M.; Trono, Didier (12 April 1996). "In Vivo Gene Delivery and Stable Transduction of Nondividing Cells by a Lentiviral Vector". Science. 272 (5259): 263–267. Bibcode:1996Sci...272..263N. doi:10.1126/science.272.5259.263. ISSN 0036-8075. PMID 8602510. S2CID 18997464.
Ibrahim M (14 July 2021). "Gene therapy restores missing dopamine in children with rare brain disease". Science. Retrieved 18 July 2021.

sciencedaily.com (Global: 993^rd place; English: 920^th place)

"Gene Therapy Corrects Sickle Cell Disease In Laboratory Study". St. Jude Children's Research Hospital. 4 December 2008. Retrieved 29 December 2012 – via ScienceDaily.
"Gene therapy cure for children with 'bubble baby' disease". Science Daily. 18 November 2014.
"Gene therapy provides safe, long-term relief for patients with severe hemophilia B". Science Daily. 20 November 2014.
"Why Gene Therapy Caused Leukemia In Some 'Boy In The Bubble Syndrome' Patients". ScienceDaily. Retrieved 19 July 2021.
"Novel gene therapy could reduce bleeding risk for haemophilia patients". ScienceDaily. Retrieved 3 August 2022.

sciencedirect.com (Global: 149^th place; English: 178^th place)

Song, Xiangrong; Liu, Chao; Wang, Ning; Huang, Hai; He, Siyan; Gong, Changyang; Wei, Yuquan (1 January 2021). "Delivery of CRISPR/Cas systems for cancer gene therapy and immunotherapy". Advanced Drug Delivery Reviews. Delivery of Biomacromolecules for Therapeutic Genome Editing. 168: 158–180. doi:10.1016/j.addr.2020.04.010. ISSN 0169-409X. PMID 32360576. S2CID 218493473.
Mennati, Afsaneh; Rostamizadeh, Kobra; Fathi, Mojtaba (1 March 2025). "Dual silencing of integrin αvβ3 receptor and insulin-like growth factor 1 receptor using mPEG-PCL/DDAB hybrid nanoparticle loaded siRNA in breast cancer therapy: An in vitro study on MCF-7 cells". International Journal of Biological Macromolecules. 294 139334. doi:10.1016/j.ijbiomac.2024.139334. ISSN 0141-8130. PMID 39743068.

semanticscholar.org (Global: 11^th place; English: 8^th place)

api.semanticscholar.org

Sheridan C (February 2011). "Gene therapy finds its niche". Nature Biotechnology. 29 (2): 121–128. doi:10.1038/nbt.1769. PMID 21301435. S2CID 5063701.
Fischer A, Hacein-Bey-Abina S, Cavazzana-Calvo M (June 2010). "20 years of gene therapy for SCID". Nature Immunology. 11 (6): 457–460. doi:10.1038/ni0610-457. PMID 20485269. S2CID 11300348.
Ferrua F, Brigida I, Aiuti A (December 2010). "Update on gene therapy for adenosine deaminase-deficient severe combined immunodeficiency". Current Opinion in Allergy and Clinical Immunology. 10 (6): 551–556. doi:10.1097/ACI.0b013e32833fea85. PMID 20966749. S2CID 205435278.
Cartier N, Aubourg P (July 2010). "Hematopoietic stem cell transplantation and hematopoietic stem cell gene therapy in X-linked adrenoleukodystrophy". Brain Pathology. 20 (4): 857–862. doi:10.1111/j.1750-3639.2010.00394.x. PMC 8094635. PMID 20626747. S2CID 24182017.
LeWitt PA, Rezai AR, Leehey MA, Ojemann SG, Flaherty AW, Eskandar EN, et al. (April 2011). "AAV2-GAD gene therapy for advanced Parkinson's disease: a double-blind, sham-surgery controlled, randomised trial". The Lancet. Neurology. 10 (4): 309–319. doi:10.1016/S1474-4422(11)70039-4. PMID 21419704. S2CID 37154043.
Pezzoli D, Chiesa R, De Nardo L, Candiani G (September 2012). "We still have a long way to go to effectively deliver genes!". Journal of Applied Biomaterials & Functional Materials. 10 (2): 82–91. doi:10.5301/JABFM.2012.9707. PMID 23015375. S2CID 6283455.
Urnov FD, Rebar EJ, Holmes MC, Zhang HS, Gregory PD (September 2010). "Genome editing with engineered zinc finger nucleases". Nature Reviews Genetics. 11 (9): 636–646. doi:10.1038/nrg2842. PMID 20717154. S2CID 205484701.
Bak RO, Gomez-Ospina N, Porteus MH (August 2018). "Gene Editing on Center Stage". Trends in Genetics. 34 (8): 600–611. doi:10.1016/j.tig.2018.05.004. PMID 29908711. S2CID 49269023.
"First CRISPR therapy dosed". Nature Biotechnology. 38 (4): 382. 1 April 2020. doi:10.1038/s41587-020-0493-4. ISSN 1546-1696. PMID 32265555. S2CID 215406440.
Mullard A (June 2020). "Gene-editing pipeline takes off". Nature Reviews. Drug Discovery. 19 (6): 367–372. doi:10.1038/d41573-020-00096-y. PMID 32415249. S2CID 218657910.
Song, Xiangrong; Liu, Chao; Wang, Ning; Huang, Hai; He, Siyan; Gong, Changyang; Wei, Yuquan (1 January 2021). "Delivery of CRISPR/Cas systems for cancer gene therapy and immunotherapy". Advanced Drug Delivery Reviews. Delivery of Biomacromolecules for Therapeutic Genome Editing. 168: 158–180. doi:10.1016/j.addr.2020.04.010. ISSN 0169-409X. PMID 32360576. S2CID 218493473.
Ginn SL, Amaya AK, Alexander IE, Edelstein M, Abedi MR (May 2018). "Gene therapy clinical trials worldwide to 2017: An update". The Journal of Gene Medicine. 20 (5) e3015. doi:10.1002/jgm.3015. PMID 29575374. S2CID 4966300.
Yin H, Kanasty RL, Eltoukhy AA, Vegas AJ, Dorkin JR, Anderson DG (August 2014). "Non-viral vectors for gene-based therapy". Nature Reviews. Genetics. 15 (8): 541–555. doi:10.1038/nrg3763. PMID 25022906. S2CID 15273455.
Singh V, Khan N, Jayandharan GR (May 2022). "Vector engineering, strategies and targets in cancer gene therapy". Cancer Gene Therapy. 29 (5): 402–417. doi:10.1038/s41417-021-00331-7. PMID 33859378. S2CID 233258994.
Anguela XM, High KA (January 2019). "Entering the Modern Era of Gene Therapy". Annual Review of Medicine. 70 (1): 273–288. doi:10.1146/annurev-med-012017-043332. PMID 30477394. S2CID 53750215.
Woods NB, Bottero V, Schmidt M, von Kalle C, Verma IM (April 2006). "Gene therapy: therapeutic gene causing lymphoma". Nature. 440 (7088): 1123. Bibcode:2006Natur.440.1123W. doi:10.1038/4401123a. PMID 16641981. S2CID 4372110.
Thrasher AJ, Gaspar HB, Baum C, Modlich U, Schambach A, Candotti F, et al. (September 2006). "Gene therapy: X-SCID transgene leukaemogenicity". Nature. 443 (7109): E5-6, discussion E6-7. Bibcode:2006Natur.443E...5T. doi:10.1038/nature05219. PMID 16988659. S2CID 4301764.
Roco MC, Bainbridge WS (2002). "Converging Technologies for Improving Human Performance: Integrating From the Nanoscale". Journal of Nanoparticle Research. 4 (4): 281–295. Bibcode:2002JNR.....4..281R. doi:10.1023/A:1021152023349. S2CID 136290217.
Allhoff F (March 2005). "Germ-line genetic enhancement and Rawlsian primary goods" (PDF). Kennedy Institute of Ethics Journal. 15 (1): 39–56. CiteSeerX 10.1.1.566.171. doi:10.1353/ken.2005.0007. PMID 15881795. S2CID 14432440. Archived (PDF) from the original on 30 January 2012.
Friedmann T, Roblin R (March 1972). "Gene therapy for human genetic disease?". Science. 175 (4025): 949–955. Bibcode:1972Sci...175..949F. doi:10.1126/science.175.4025.949. PMID 5061866. S2CID 19952096.
Cepko CL, Roberts BE, Mulligan RC (July 1984). "Construction and applications of a highly transmissible murine retrovirus shuttle vector. This Vector is used for entering a cell in the humans cell body". Cell. 37 (3): 1053–1062. doi:10.1016/0092-8674(84)90440-9. PMID 6331674. S2CID 34544709.
Blaese RM, Culver KW, Miller AD, Carter CS, Fleisher T, Clerici M, et al. (October 1995). "T lymphocyte-directed gene therapy for ADA- SCID: initial trial results after 4 years". Science. 270 (5235): 475–480. Bibcode:1995Sci...270..475B. doi:10.1126/science.270.5235.475. PMID 7570001. S2CID 46339645.
Abbott A (April 1992). "Gene therapy. Italians first to use stem cells". Nature. 356 (6369): 465. Bibcode:1992Natur.356..465A. doi:10.1038/356465a0. PMID 1560817. S2CID 4319842.
Cavazzana-Calvo M, Thrasher A, Mavilio F (February 2004). "The future of gene therapy". Nature. 427 (6977): 779–781. Bibcode:2004Natur.427..779C. doi:10.1038/427779a. hdl:11380/307566. PMID 14985734. S2CID 4421364.
Naldini, Luigi; Blömer, Ulrike; Gallay, Philippe; Ory, Daniel; Mulligan, Richard; Gage, Fred H.; Verma, Inder M.; Trono, Didier (12 April 1996). "In Vivo Gene Delivery and Stable Transduction of Nondividing Cells by a Lentiviral Vector". Science. 272 (5259): 263–267. Bibcode:1996Sci...272..263N. doi:10.1126/science.272.5259.263. ISSN 0036-8075. PMID 8602510. S2CID 18997464.
Ott MG, Schmidt M, Schwarzwaelder K, Stein S, Siler U, Koehl U, et al. (April 2006). "Correction of X-linked chronic granulomatous disease by gene therapy, augmented by insertional activation of MDS1-EVI1, PRDM16 or SETBP1". Nature Medicine. 12 (4): 401–409. doi:10.1038/nm1393. PMID 16582916. S2CID 7601162.
Brown BD, Venneri MA, Zingale A, Sergi Sergi L, Naldini L (May 2006). "Endogenous microRNA regulation suppresses transgene expression in hematopoietic lineages and enables stable gene transfer". Nature Medicine. 12 (5): 585–591. doi:10.1038/nm1398. PMID 16633348. S2CID 11114427.
Cartier N, Hacein-Bey-Abina S, Bartholomae CC, Veres G, Schmidt M, Kutschera I, et al. (November 2009). "Hematopoietic stem cell gene therapy with a lentiviral vector in X-linked adrenoleukodystrophy" (PDF). Science. 326 (5954): 818–823. Bibcode:2009Sci...326..818C. doi:10.1126/science.1171242. PMID 19892975. S2CID 27783.
Yang ZJ, Zhang YR, Chen B, Zhang SL, Jia EZ, Wang LS, et al. (July 2009). "Phase I clinical trial on intracoronary administration of Ad-hHGF treating severe coronary artery disease". Molecular Biology Reports. 36 (6): 1323–1329. doi:10.1007/s11033-008-9315-3. PMID 18649012. S2CID 23419866.
Hahn W, Pyun WB, Kim DS, Yoo WS, Lee SD, Won JH, et al. (October 2011). "Enhanced cardioprotective effects by coexpression of two isoforms of hepatocyte growth factor from naked plasmid DNA in a rat ischemic heart disease model". The Journal of Gene Medicine. 13 (10): 549–555. doi:10.1002/jgm.1603. PMID 21898720. S2CID 26812780.
Deev RV, Bozo IY, Mzhavanadze ND, Voronov DA, Gavrilenko AV, Chervyakov YV, et al. (September 2015). "pCMV-vegf165 Intramuscular Gene Transfer is an Effective Method of Treatment for Patients With Chronic Lower Limb Ischemia". Journal of Cardiovascular Pharmacology and Therapeutics. 20 (5): 473–482. doi:10.1177/1074248415574336. PMID 25770117. S2CID 13443907.
Morrison C (3 March 2015). "$1-million price tag set for Glybera gene therapy". TradeSecrets. 33 (3): 217–218. doi:10.1038/nbt0315-217. PMID 25748892. S2CID 205266596.
Fernàndez-Ruiz I (March 2016). "Gene therapy: No improvement in outcomes with gene therapy for heart failure". Nature Reviews. Cardiology. 13 (3): 122–123. doi:10.1038/nrcardio.2016.14. PMID 26843287. S2CID 205472001.
Biffi A, Montini E, Lorioli L, Cesani M, Fumagalli F, Plati T, et al. (August 2013). "Lentiviral hematopoietic stem cell gene therapy benefits metachromatic leukodystrophy". Science. 341 (6148) 1233158. doi:10.1126/science.1233158. PMID 23845948. S2CID 206546808.
Ledford H (5 September 2018). "First test of in-body gene editing shows promise". Nature. doi:10.1038/d41586-018-06195-6. S2CID 92840885.
"High-dose AAV gene therapy deaths". Nature Biotechnology. 38 (8): 910. 1 August 2020. doi:10.1038/s41587-020-0642-9. ISSN 1546-1696. PMID 32760031. S2CID 220981004.
Gillmore JD, Gane E, Taubel J, Kao J, Fontana M, Maitland ML, et al. (August 2021). "CRISPR-Cas9 In Vivo Gene Editing for Transthyretin Amyloidosis". The New England Journal of Medicine. 385 (6): 493–502. doi:10.1056/NEJMoa2107454. PMID 34215024. S2CID 235722446.
"The science news that shaped 2021: Nature's picks". Nature. December 2021. doi:10.1038/d41586-021-03734-6. PMID 34907370. S2CID 245144020.
Flotte TR, Cataltepe O, Puri A, Batista AR, Moser R, McKenna-Yasek D, et al. (February 2022). "AAV gene therapy for Tay-Sachs disease". Nature Medicine. 28 (2): 251–259. doi:10.1038/s41591-021-01664-4. PMC 10786171. PMID 35145305. S2CID 246748772.
Chowdary P, Shapiro S, Makris M, Evans G, Boyce S, Talks K, et al. (July 2022). "Phase 1-2 Trial of AAVS3 Gene Therapy in Patients with Hemophilia B". The New England Journal of Medicine. 387 (3): 237–247. doi:10.1056/NEJMoa2119913. PMID 35857660. S2CID 250697905.

singularityhub.com (Global: low place; English: low place)

"Gene Therapy Turns Several Leukemia Patients Cancer Free. Will It Work for Other Cancers, Too?". Singularity Hub. 6 January 2014. Retrieved 7 January 2014.

smithsonianmag.com (Global: 503^rd place; English: 364^th place)

Pittman J, Ravitz JD. "These Scientists May Have Found a Cure for 'Bubble Boy' Disease". Smithsonian Magazine. Retrieved 19 July 2021.

statnews.com (Global: 8,771^st place; English: 5,253^rd place)

Marchione M (11 May 2021). "AIDS virus used in gene therapy to fix 'bubble baby' disease". Stat. Retrieved 19 July 2021.
"Gene therapy trial points to a wider window to alter course of rare disease". Stat. 12 July 2021. Retrieved 18 July 2021.

technologyreview.com (Global: 1,943^rd place; English: 1,253^rd place)

Rojahn SY (11 February 2014). "Genome Surgery". MIT Technology Review. Archived from the original on 15 February 2014. Retrieved 17 February 2014.
Regalado A (4 May 2016). "The World's Most Expensive Medicine Is a Bust". MIT Technology Review.

the-scientist.com (Global: 7,827^th place; English: 5,905^th place)

Richards S (6 November 2012). "Gene Therapy Arrives in Europe". The Scientist.
Wilson JF (18 March 2002). "Murine Gene Therapy Corrects Symptoms of Sickle Cell Disease". The Scientist – Magazine of the Life Sciences. Archived from the original on 1 April 2020. Retrieved 17 August 2010.

theatlantic.com (Global: 228^th place; English: 158^th place)

Zhang S (15 November 2017). "The First Man to Have His Genes Edited Inside His Body". The Atlantic. Retrieved 17 November 2020.

theconversation.com (Global: 928^th place; English: 651^st place)

Sena-Esteves M (14 February 2022). "First gene therapy for Tay-Sachs disease successfully given to two children". The Conversation. Retrieved 7 March 2022.

theguardian.com (Global: 12^th place; English: 11^th place)

Bosely S (30 April 2013). "Pioneering gene therapy trials offer hope for heart patients". The Guardian. Retrieved 28 April 2014.
Sample I (5 November 2015). "Baby girl is first in the world to be treated with 'designer immune cells'". The Guardian. Retrieved 6 November 2015.

thepharmaletter.com (Global: low place; English: low place)

"Chiesi and uniQure delay Glybera launch to add data". Biotechnology. The Pharma Letter. 4 August 2014. Retrieved 28 August 2014.

thephysiciansclinic.co.uk (Global: low place; English: low place)

"Cardiac gene therapy news: The Physicians Clinic". thephysiciansclinic.co.uk. 8 September 2013. Archived from the original on 29 April 2014. Retrieved 2 January 2022.

thetimes.com (Global: low place; English: low place)

Henry R (19 February 2017). "Leukaemia cure hopes rise as girl is gene‑edited". The Times. Retrieved 20 February 2017.
Whipple T (1 March 2017). "New gene therapy 'shrinks tumours like ice cubes'". The Times. Retrieved 1 March 2017.

ucl.ac.uk (Global: 1,306^th place; English: 885^th place)

discovery.ucl.ac.uk

Chowdary P, Shapiro S, Makris M, Evans G, Boyce S, Talks K, et al. (July 2022). "Phase 1-2 Trial of AAVS3 Gene Therapy in Patients with Hemophilia B". The New England Journal of Medicine. 387 (3): 237–247. doi:10.1056/NEJMoa2119913. PMID 35857660. S2CID 250697905.

uniqure.com (Global: low place; English: low place)

"First Gene Therapy Approved by European Commission". UniQure (Press release). 2 November 2012. Archived from the original on 5 November 2012. Retrieved 2 January 2022.

utah.edu (Global: 2,446^th place; English: 1,661^st place)

learn.genetics.utah.edu

"Challenges In Gene Therapy". learn.genetics.utah.edu. Retrieved 23 July 2021.

wada-ama.org (Global: 9,542^nd place; English: 8,313^th place)

"WADA Gene Doping". World Anti-Doping Agency. WADA. Archived from the original on 21 November 2009. Retrieved 27 September 2013.

wasdarwinwrong.com (Global: low place; English: low place)

Korthof G. "The implications of Steele's soma-to-germline feedback for human gene therapy".

washingtonpost.com (Global: 34^th place; English: 27^th place)

Stein R (11 October 2010). "First patient treated in stem cell study". The Washington Post. Retrieved 10 November 2010.

web.archive.org (Global: 1^st place; English: 1^st place)

"What is Gene Therapy?". U.S. Food and Drug Administration (FDA). 9 December 2020. Archived from the original on 7 June 2019.
"Gene Therapy Clinical Trials Worldwide Database". The Journal of Gene Medicine. Wiley. June 2016. Archived from the original on 31 July 2020.
"What is gene therapy?". Genetics Home Reference. 28 March 2016. Archived from the original on 6 April 2016. Retrieved 2 January 2022.
"International Law". The Genetics and Public Policy Center, Johns Hopkins University Berman Institute of Bioethics. 2010. Archived from the original on 2 September 2014.
"Human Cloning and Genetic Modification". Association of Reproductive Health Officials. 2013. Archived from the original on 18 June 2013.
"Gene Therapy". ama-assn.org. 4 April 2014. Archived from the original on 15 March 2015. Retrieved 22 March 2015.
"Gene & Cell Therapy FAQs | ASGCT - American Society of Gene & Cell Therapy | ASGCT - American Society of Gene & Cell Therapy". asgct.org. Archived from the original on 23 July 2021. Retrieved 23 July 2021.
"FDA Approves First Gene Therapy for Children with Metachromatic Leukodystrophy". U.S. Food and Drug Administration (FDA) (Press release). 9 August 2024. Archived from the original on 18 March 2024.
"FDA Approves First Topical Gene Therapy for Treatment of Wounds in Patients with Dystrophic Epidermolysis Bullosa". U.S. Food and Drug Administration (FDA) (Press release). 19 May 2023. Archived from the original on 19 May 2023. Retrieved 31 July 2023.
"FDA Approves First Cell-Based Gene Therapy to Treat Adult and Pediatric Patients with Beta-thalassemia Who Require Regular Blood Transfusions". U.S. Food and Drug Administration (FDA) (Press release). 17 August 2022. Archived from the original on 17 August 2022. Retrieved 31 August 2022.
"FDA approves brexucabtagene autoleucel for relapsed or refractory B-cell precursor acute lymphoblastic leukemia". U.S. Food and Drug Administration (FDA). 1 October 2021. Archived from the original on 2 October 2021. Retrieved 2 October 2021.
"FDA Approves First Gene Therapy for Treatment of Certain Patients with Duchenne Muscular Dystrophy". U.S. Food and Drug Administration (FDA) (Press release). 23 June 2023. Archived from the original on 22 June 2023. Retrieved 12 July 2023.
"FDA Approves First Gene Therapy for Treatment of Aromatic L-amino Acid Decarboxylase Deficiency". U.S. Food and Drug Administration (FDA) (Press release). 14 November 2024. Archived from the original on 14 November 2024. Retrieved 17 November 2024. This article incorporates text from this source, which is in the public domain.
"Upstaza EPAR". European Medicines Agency (EMA). 13 April 2022. Archived from the original on 14 March 2023. Retrieved 3 March 2023. Text was copied from this source which is copyright European Medicines Agency. Reproduction is authorized provided the source is acknowledged.
"Casgevy". U.S. Food and Drug Administration (FDA). 8 December 2023. Archived from the original on 8 December 2023. Retrieved 8 December 2023.
"Abecma (idecabtagene vicleucel)". U.S. Food and Drug Administration (FDA). 21 April 2021. Archived from the original on 17 May 2021.
"Breyanzi (lisocabtagene maraleucel)". U.S. Food and Drug Administration (FDA). 4 March 2021. Archived from the original on 28 February 2021.
"Lyfgenia". U.S. Food and Drug Administration (FDA). 8 December 2023. Archived from the original on 8 December 2023. Retrieved 9 December 2023.
"FDA Approves First Gene Therapy for the Treatment of High-Risk, Non-Muscle-Invasive Bladder Cancer". U.S. Food and Drug Administration (FDA) (Press release). 16 December 2022. Archived from the original on 16 December 2022. Retrieved 16 December 2022.
"FDA approves obecabtagene autoleucel for acute lymphoblastic leukemia". U.S. Food and Drug Administration (FDA). 8 November 2024. Archived from the original on 8 November 2024. Retrieved 10 November 2024. This article incorporates text from this source, which is in the public domain.
"Zolgensma". U.S. Food and Drug Administration (FDA). 16 March 2021. Archived from the original on 12 September 2019.
"EMA-approved application for Zolgensma". Aetion (Press release). Archived from the original on 23 July 2021. Retrieved 23 July 2021.
"First gene therapy to treat severe haemophilia A". European Medicines Agency (EMA) (Press release). 24 June 2022. Archived from the original on 26 June 2022. Retrieved 26 June 2022.
"Roctavian: Pending EC decision". European Medicines Agency (EMA). 23 June 2022. Archived from the original on 26 June 2022. Retrieved 26 June 2022.
"FDA approves novel gene therapy to treat patients with a rare form of inherited vision loss". U.S. Food and Drug Administration (FDA) (Press release). 24 March 2020. Archived from the original on 26 May 2019. Retrieved 23 July 2021.
Rojahn SY (11 February 2014). "Genome Surgery". MIT Technology Review. Archived from the original on 15 February 2014. Retrieved 17 February 2014.
"Gene Therapy". Oak Ridge National Laboratory. 20 December 2012. Archived from the original on 20 December 2012. Retrieved 2 January 2022.
HUGO Ethics Committee (April 2001). "Statement on Gene Therapy Research" (PDF). Human Genome Organization. Archived from the original (PDF) on 7 September 2008.
Isasi RM, Nguyen TM, Knoppers BM (October 2006). "National Regulatory Frameworks Regarding Human Genetic Modification Technologies (Somatic and Germline Modification)" (PDF). Genetics & Public Policy Center. Archived from the original on 17 September 2014.
"NIH Guidelines for Research Involving Recombinant or Synthetic Nucleic Acid Molecules". U.S. National Institutes of Health. April 2016. Archived from the original on 28 May 2016.
U.S. Food and Drug Administration (14 October 1993). "Application of Current Statutory Authorities to Human Somatic Cell Therapy Products and Gene Therapy Products" (PDF). Federal Register. 58 (197). Archived from the original (PDF) on 21 June 2009.
"Guidance for Industry: Guidance for Human Somatic Cell Therapy and Gene Therapy". U.S. Food and Drug Administration (FDA). 9 July 2009. Archived from the original on 9 July 2009. Retrieved 2 January 2022.
"WADA Gene Doping". World Anti-Doping Agency. WADA. Archived from the original on 21 November 2009. Retrieved 27 September 2013.
"The Center for Health Ethics - University of Missouri School of Medicine - Gene Therapy and Genetic Engineering". ethics.missouri.edu. 3 December 2013. Archived from the original on 3 December 2013. Retrieved 2 January 2022.
Allhoff F (March 2005). "Germ-line genetic enhancement and Rawlsian primary goods" (PDF). Kennedy Institute of Ethics Journal. 15 (1): 39–56. CiteSeerX 10.1.1.566.171. doi:10.1353/ken.2005.0007. PMID 15881795. S2CID 14432440. Archived (PDF) from the original on 30 January 2012.
"The Declaration of Inuyama: Human Genome Mapping, Genetic Screening and Gene Therapy". Council for International Organizations of Medical Sciences. Archived from the original on 5 August 2001.
"The first gene therapy". Life Sciences Foundation. 21 June 2011. Archived from the original on 28 November 2012. Retrieved 7 January 2014.
Wilson JF (18 March 2002). "Murine Gene Therapy Corrects Symptoms of Sickle Cell Disease". The Scientist – Magazine of the Life Sciences. Archived from the original on 1 April 2020. Retrieved 17 August 2010.
"Penn Medicine presents HIV gene therapy trial data at CROI 2009". EurekAlert!. 10 February 2009. Archived from the original on 25 August 2012. Retrieved 19 November 2009.
"On Cancer: Launch of Stem Cell Therapy Trial Offers Hope for Patients with Inherited Blood Disorder". Memorial Sloan Kettering Cancer Center. Memorial Sloan-Kettering Cancer Center. 16 July 2012. Archived from the original on 8 June 2020. Retrieved 15 December 2012.
"First Gene Therapy Approved by European Commission". UniQure (Press release). 2 November 2012. Archived from the original on 5 November 2012. Retrieved 2 January 2022.
"Gene therapy approved in Europe for first time". Archived from the original on 4 January 2014.
"Cardiac gene therapy news: The Physicians Clinic". thephysiciansclinic.co.uk. 8 September 2013. Archived from the original on 29 April 2014. Retrieved 2 January 2022.
"Celladon Receives Breakthrough Therapy Designation From FDA for Mydicar, Novel, First-in-Class Therapy in Development to Treat Heart Failure". Celladon. 10 July 2015. Archived from the original on 10 July 2015. Retrieved 2 January 2022.
"Summary of opinion1 (initial authorisation) Strimvelis" (PDF). European Medicines Agency. 1 April 2016. pp. 1–2. Archived from the original (PDF) on 28 January 2018. Retrieved 13 April 2016.
Reeves R (6 June 2016). "Second gene therapy wins approval in Europe". Bionews. Archived from the original on 21 February 2017. Retrieved 20 February 2017.
"FDA approval brings first gene therapy to the United States" (Press release). U.S. Food and Drug Administration (FDA). 30 August 2017. Archived from the original on 13 May 2019.
"FDA approves axicabtagene ciloleucel for large B-cell lymphoma". U.S. Food and Drug Administration (FDA). Archived from the original on 12 June 2019. Retrieved 5 January 2018.
Zayner J (6 October 2017). "DIY Human CRISPR Myostatin Knock-Out". Youtube. Archived from the original on 11 November 2017. Retrieved 20 May 2020.
"FDA approves novel gene therapy to treat patients with a rare form of inherited vision loss". U.S. Food and Drug Administration (FDA) (Press release). 19 December 2017. Archived from the original on 26 May 2019. Retrieved 20 December 2017.
"Audentes Therapeutics Provides Update on the ASPIRO Clinical Trial Evaluating AT132 in Patients with X-linked Myotubular Myopathy". Audentes Therapeutics. 10 August 2020. Archived from the original on 27 October 2020. Retrieved 21 September 2020.
"Libmeldy: Pending EC decision". European Medicines Agency (EMA). 16 October 2020. Archived from the original on 17 October 2020. Retrieved 16 October 2020. Text was copied from this source which is copyright European Medicines Agency. Reproduction is authorized provided the source is acknowledged.
Lysogene (15 October 2020). "Lysogene provides update on the AAVance Clinical Trial Evaluating LYS-SAF302 in Patients with MPS IIIA – Lysogene". Archived from the original on 17 October 2020. Retrieved 17 October 2020.
"Upstaza: Pending EC decision". European Medicines Agency. 19 May 2022. Archived from the original on 25 May 2022. Retrieved 22 May 2022.
"PTC Therapeutics Receives Positive CHMP Opinion for Upstaza for the Treatment of AADC Deficiency". PTC Therapeutics (Press release). 20 May 2022. Archived from the original on 23 May 2022. Retrieved 22 May 2022.
"PTC Therapeutics Announces FDA Approval of AADC Deficiency Gene Therapy". PTC Therapeutics, Inc. 13 November 2024. Archived from the original on 15 November 2024. Retrieved 21 November 2024.

whatisbiotechnology.org (Global: low place; English: low place)

"Gene therapy". WhatIsBiotechnology.org. The Biotechnology and Medicine Education Trust (Biotechmet). Retrieved 7 July 2023.

who.int (Global: 195^th place; English: 302^nd place)

"Human genome editing: recommendations". www.who.int. Retrieved 21 April 2025.

wiley.com (Global: 222^nd place; English: 297^th place)

onlinelibrary.wiley.com

Ranisch R (January 2020). "Special Issue:Human Germline Editing". Bioethics. 34 (1): 1–143.

worldcat.org (Global: 5^th place; English: 5^th place)

search.worldcat.org

"First CRISPR therapy dosed". Nature Biotechnology. 38 (4): 382. 1 April 2020. doi:10.1038/s41587-020-0493-4. ISSN 1546-1696. PMID 32265555. S2CID 215406440.
Nóbrega C, Mendonça L, Matos CA (2020). A handbook of gene and cell therapy. Cham: Springer. ISBN 978-3-030-41333-0. OCLC 1163431307.
Song, Xiangrong; Liu, Chao; Wang, Ning; Huang, Hai; He, Siyan; Gong, Changyang; Wei, Yuquan (1 January 2021). "Delivery of CRISPR/Cas systems for cancer gene therapy and immunotherapy". Advanced Drug Delivery Reviews. Delivery of Biomacromolecules for Therapeutic Genome Editing. 168: 158–180. doi:10.1016/j.addr.2020.04.010. ISSN 0169-409X. PMID 32360576. S2CID 218493473.
Jensen, Thomas Leth; Gøtzsche, Casper René; Woldbye, David P. D. (2021). "Current and Future Prospects for Gene Therapy for Rare Genetic Diseases Affecting the Brain and Spinal Cord". Frontiers in Molecular Neuroscience. 14 695937. doi:10.3389/fnmol.2021.695937. ISSN 1662-5099. PMC 8527017. PMID 34690692.
Krasilnikova, O.; Yakimova, A.; Ivanov, S.; Atiakshin, D.; Kostin, A.A.; Sosin, D.; Shegay, P.; Kaprin, A.D.; Klabukov, I. (2023). "Gene-Activated Materials in Regenerative Dentistry: Narrative Review of Technology and Study Results". International Journal of Molecular Sciences. 24 (22) 16250. doi:10.3390/ijms242216250. ISSN 1422-0067. PMC 10671237. PMID 38003439.
Foldvari, M.; Chen, D.W.; Nafissi, N.; Calderon, D.; Narsineni, L.; Rafiee, A. (2016). "Non-viral gene therapy: Gains and challenges of non-invasive administration methods". Journal of Controlled Release. 240: 165–190. doi:10.1016/j.jconrel.2015.12.012. ISSN 1873-4995. PMID 26686079.
Mennati, Afsaneh; Rostamizadeh, Kobra; Fathi, Mojtaba (1 March 2025). "Dual silencing of integrin αvβ3 receptor and insulin-like growth factor 1 receptor using mPEG-PCL/DDAB hybrid nanoparticle loaded siRNA in breast cancer therapy: An in vitro study on MCF-7 cells". International Journal of Biological Macromolecules. 294 139334. doi:10.1016/j.ijbiomac.2024.139334. ISSN 0141-8130. PMID 39743068.
Keam, Susan J. (November 2024). "Afamitresgene Autoleucel: First Approval". Molecular Diagnosis & Therapy. 28 (6): 861–866. doi:10.1007/s40291-024-00749-3. ISSN 1179-2000. PMID 39404764.
Scott, Lesley J. (February 2015). "Alipogene tiparvovec: a review of its use in adults with familial lipoprotein lipase deficiency". Drugs. 75 (2): 175–182. doi:10.1007/s40265-014-0339-9. ISSN 1179-1950. PMID 25559420.
Guo, Congting; Ma, Xiaoteng; Gao, Fei; Guo, Yuxuan (9 March 2023). "Off-target effects in CRISPR/Cas9 gene editing". Frontiers in Bioengineering and Biotechnology. 11 1143157. doi:10.3389/fbioe.2023.1143157. ISSN 2296-4185. PMC 10034092. PMID 36970624.
Wu, Shao-Shuai; Li, Qing-Cui; Yin, Chang-Qing; Xue, Wen; Song, Chun-Qing (15 March 2020). "Advances in CRISPR/Cas-based Gene Therapy in Human Genetic Diseases". Theranostics. 10 (10): 4374–4382. doi:10.7150/thno.43360. ISSN 1838-7640. PMC 7150498. PMID 32292501.
Harmon A (14 February 2017). "Human Gene Editing Receives Science Panel's Support". The New York Times. ISSN 0362-4331. Archived from the original on 2 January 2022. Retrieved 17 February 2017.
Blakeslee S (18 May 1993). "Treatment for 'Bubble Boy Disease'". The New York Times. ISSN 0362-4331. Retrieved 9 February 2018.
Naldini, Luigi; Blömer, Ulrike; Gallay, Philippe; Ory, Daniel; Mulligan, Richard; Gage, Fred H.; Verma, Inder M.; Trono, Didier (12 April 1996). "In Vivo Gene Delivery and Stable Transduction of Nondividing Cells by a Lentiviral Vector". Science. 272 (5259): 263–267. Bibcode:1996Sci...272..263N. doi:10.1126/science.272.5259.263. ISSN 0036-8075. PMID 8602510. S2CID 18997464.
"High-dose AAV gene therapy deaths". Nature Biotechnology. 38 (8): 910. 1 August 2020. doi:10.1038/s41587-020-0642-9. ISSN 1546-1696. PMID 32760031. S2CID 220981004.

wsj.com (Global: 79^th place; English: 65^th place)

Whalen J (2 November 2012). "Gene-Therapy Approval Marks Major Milestone". The Wall Street Journal.

youtube.com (Global: 9^th place; English: 13^th place)

Zayner J (6 October 2017). "DIY Human CRISPR Myostatin Knock-Out". Youtube. Archived from the original on 11 November 2017. Retrieved 20 May 2020.

zenodo.org (Global: 621^st place; English: 380^th place)

Blaese RM, Culver KW, Miller AD, Carter CS, Fleisher T, Clerici M, et al. (October 1995). "T lymphocyte-directed gene therapy for ADA- SCID: initial trial results after 4 years". Science. 270 (5235): 475–480. Bibcode:1995Sci...270..475B. doi:10.1126/science.270.5235.475. PMID 7570001. S2CID 46339645.

Gene therapy (English Wikipedia)

aabb.org (Global: low place; English: low place)

abedia.com (Global: low place; English: low place)

aetion.com (Global: low place; English: low place)

allfordrugs.com (Global: low place; English: low place)

ama-assn.org (Global: 3,257th place; English: 3,398th place)

apnews.com (Global: 129th place; English: 89th place)

archive.org (Global: 6th place; English: 6th place)

archives-ouvertes.fr (Global: 1,031st place; English: 879th place)

hal-cea.archives-ouvertes.fr

arhp.org (Global: low place; English: low place)

asgct.org (Global: low place; English: low place)

audentestx.com (Global: low place; English: low place)

bbc.co.uk (Global: 8th place; English: 10th place)

bbc.co.uk

news.bbc.co.uk

bbc.com (Global: 20th place; English: 30th place)

bionews.org.uk (Global: low place; English: low place)

books.google.com (Global: 3rd place; English: 3rd place)

bostonglobe.com (Global: 553rd place; English: 334th place)

cancer.gov (Global: 1,684th place; English: 1,350th place)

celladon.com (Global: low place; English: low place)

ir.celladon.com

cioms.ch (Global: low place; English: low place)

clinicaltrials.gov (Global: 5,225th place; English: 4,062nd place)

crisprmedicinenews.com (Global: low place; English: low place)

dddmag.com (Global: low place; English: low place)

doi.org (Global: 2nd place; English: 2nd place)

editasmedicine.com (Global: low place; English: low place)

ir.editasmedicine.com

endpts.com (Global: low place; English: low place)

eurekalert.org (Global: 3,538th place; English: 3,083rd place)

eurolab.ua (Global: low place; English: low place)

europa.eu (Global: 68th place; English: 117th place)

ema.europa.eu

ec.europa.eu

factcheck.org (Global: 7,075th place; English: 4,103rd place)

fda.gov (Global: 447th place; English: 338th place)

fiercebiotech.com (Global: low place; English: 8,828th place)

forbes.com (Global: 54th place; English: 48th place)

fullfact.org (Global: low place; English: low place)

genome.gov (Global: 9,308th place; English: 8,899th place)

ghostarchive.org (Global: 32nd place; English: 21st place)

globenewswire.com (Global: 2,614th place; English: 1,549th place)

handle.net (Global: 102nd place; English: 76th place)

hdl.handle.net

harvard.edu (Global: 18th place; English: 17th place)

ui.adsabs.harvard.edu

hhs.gov (Global: 5,973rd place; English: 3,693rd place)

hugo-international.org (Global: low place; English: low place)

independent.co.uk (Global: 36th place; English: 33rd place)

io9.com (Global: 2,391st place; English: 1,563rd place)

jetpress.org (Global: low place; English: low place)

josiahzayner.com (Global: low place; English: low place)

jyi.org (Global: low place; English: low place)

legacy.com (Global: 672nd place; English: 400th place)

liebertpub.com (Global: low place; English: low place)

lifesciencesfoundation.org (Global: low place; English: low place)

luxturna.com (Global: low place; English: low place)

lysogene.com (Global: low place; English: low place)

medlineplus.gov (Global: 1,581st place; English: 1,299th place)

medpagetoday.com (Global: 9,274th place; English: 5,856th place)

medscape.com (Global: 719th place; English: 636th place)

missouri.edu (Global: 3,342nd place; English: 3,036th place)

ethics.missouri.edu

mskcc.org (Global: low place; English: 7,046th place)

nationalacademies.org (Global: 7,189th place; English: 6,078th place)

nature.com (Global: 234th place; English: 397th place)

newscientist.com (Global: 774th place; English: 716th place)

newsweek.com (Global: 269th place; English: 201st place)

nih.gov (Global: 4th place; English: 4th place)

pubmed.ncbi.nlm.nih.gov

ncbi.nlm.nih.gov

nih.gov

osp.od.nih.gov

oar.nih.gov

npr.org (Global: 92nd place; English: 72nd place)

nymag.com (Global: 346th place; English: 229th place)

nypost.com (Global: 208th place; English: 156th place)

nytimes.com (Global: 7th place; English: 7th place)

ama-assn.org (Global: 3,257^th place; English: 3,398^th place)

apnews.com (Global: 129^th place; English: 89^th place)

archive.org (Global: 6^th place; English: 6^th place)

archives-ouvertes.fr (Global: 1,031^st place; English: 879^th place)

bbc.co.uk (Global: 8^th place; English: 10^th place)

bbc.com (Global: 20^th place; English: 30^th place)

books.google.com (Global: 3^rd place; English: 3^rd place)

bostonglobe.com (Global: 553^rd place; English: 334^th place)

cancer.gov (Global: 1,684^th place; English: 1,350^th place)

clinicaltrials.gov (Global: 5,225^th place; English: 4,062^nd place)

doi.org (Global: 2^nd place; English: 2^nd place)

eurekalert.org (Global: 3,538^th place; English: 3,083^rd place)

europa.eu (Global: 68^th place; English: 117^th place)

factcheck.org (Global: 7,075^th place; English: 4,103^rd place)

fda.gov (Global: 447^th place; English: 338^th place)

fiercebiotech.com (Global: low place; English: 8,828^th place)

forbes.com (Global: 54^th place; English: 48^th place)

genome.gov (Global: 9,308^th place; English: 8,899^th place)

ghostarchive.org (Global: 32^nd place; English: 21^st place)

globenewswire.com (Global: 2,614^th place; English: 1,549^th place)

handle.net (Global: 102^nd place; English: 76^th place)

harvard.edu (Global: 18^th place; English: 17^th place)

hhs.gov (Global: 5,973^rd place; English: 3,693^rd place)

independent.co.uk (Global: 36^th place; English: 33^rd place)

io9.com (Global: 2,391^st place; English: 1,563^rd place)

legacy.com (Global: 672^nd place; English: 400^th place)

medlineplus.gov (Global: 1,581^st place; English: 1,299^th place)

medpagetoday.com (Global: 9,274^th place; English: 5,856^th place)

medscape.com (Global: 719^th place; English: 636^th place)

missouri.edu (Global: 3,342^nd place; English: 3,036^th place)

mskcc.org (Global: low place; English: 7,046^th place)

nationalacademies.org (Global: 7,189^th place; English: 6,078^th place)

nature.com (Global: 234^th place; English: 397^th place)

newscientist.com (Global: 774^th place; English: 716^th place)

newsweek.com (Global: 269^th place; English: 201^st place)

nih.gov (Global: 4^th place; English: 4^th place)

npr.org (Global: 92^nd place; English: 72^nd place)

nymag.com (Global: 346^th place; English: 229^th place)

nypost.com (Global: 208^th place; English: 156^th place)

nytimes.com (Global: 7^th place; English: 7^th place)

ornl.gov (Global: 5,534^th place; English: 4,538^th place)

politifact.com (Global: 3,208^th place; English: 1,803^rd place)

popularmechanics.com (Global: 1,808^th place; English: 1,159^th place)

psu.edu (Global: 207^th place; English: 136^th place)

reuters.com (Global: 49^th place; English: 47^th place)

science.org (Global: 1,160^th place; English: 737^th place)

sciencedaily.com (Global: 993^rd place; English: 920^th place)

sciencedirect.com (Global: 149^th place; English: 178^th place)

semanticscholar.org (Global: 11^th place; English: 8^th place)

smithsonianmag.com (Global: 503^rd place; English: 364^th place)

statnews.com (Global: 8,771^st place; English: 5,253^rd place)

technologyreview.com (Global: 1,943^rd place; English: 1,253^rd place)

the-scientist.com (Global: 7,827^th place; English: 5,905^th place)

theatlantic.com (Global: 228^th place; English: 158^th place)

theconversation.com (Global: 928^th place; English: 651^st place)

theguardian.com (Global: 12^th place; English: 11^th place)

ucl.ac.uk (Global: 1,306^th place; English: 885^th place)

utah.edu (Global: 2,446^th place; English: 1,661^st place)

wada-ama.org (Global: 9,542^nd place; English: 8,313^th place)

washingtonpost.com (Global: 34^th place; English: 27^th place)

web.archive.org (Global: 1^st place; English: 1^st place)

who.int (Global: 195^th place; English: 302^nd place)

wiley.com (Global: 222^nd place; English: 297^th place)

worldcat.org (Global: 5^th place; English: 5^th place)

wsj.com (Global: 79^th place; English: 65^th place)

youtube.com (Global: 9^th place; English: 13^th place)

zenodo.org (Global: 621^st place; English: 380^th place)