Chirmule N, Propert K, Magosin S, Qian Y, Qian R, Wilson J (September 1999). “Immune responses to adenovirus and adeno-associated virus in humans”. Gene Therapy6 (9): 1574–83. doi:10.1038/sj.gt.3300994. PMID10490767.
Ponnazhagan S, Mukherjee P, Yoder MC, et al. (April 1997). “Adeno-associated virus 2-mediated gene transfer in vivo: organ-tropism and expression of transduced sequences in mice”. Gene190 (1): 203–10. doi:10.1016/S0378-1119(96)00576-8. PMID9185868.
McCarty, D M; Monahan, P E; Samulski, R J (2001). “Self-complementary recombinant adeno-associated virus (scAAV) vectors promote efficient transduction independently of DNA synthesis”. Gene Therapy8 (16): 1248–54. doi:10.1038/sj.gt.3301514. PMID11509958.
Maguire, A. M., Simonelli, F., Pierce, E. A., Pugh, E. N., Mingozzi, F., Bennicelli, J., Banfi, S., et al. (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
Bainbridge, J. W. B., Smith, A. J., Barker, S. S., Robbie, S., Henderson, R., Balaggan, K., Viswanathan, A., et al. (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/NEJMoa0802268PMID18441371
Hauswirth, W. W., Aleman, T. S., Kaushal, S., Cideciyan, A. V., Schwartz, S. B., Wang, L., Conlon, T. J., et al. (2008). Treatment of Leber Congenital Amaurosis Due to RPE65Mutations by Ocular Subretinal Injection of Adeno-Associated Virus Gene Vector: Short-Term Results of a Phase I Trial. Human gene therapy, 19(10), 979–990. doi:10.1089/hum.2008.107PMID18774912
Nathwani, A. C., Tuddenham, E. G. D., Rangarajan, S., Rosales, C., McIntosh, J., Linch, D. C., Chowdary, P., et al. (2011). Adenovirus-associated virus vector-mediated gene transfer in hemophilia B The New England journal of medicine, 365(25), 2357–2365. doi:10.1056/NEJMoa1108046
LeWitt, P. A., Rezai, A. R., Leehey, M. A., Ojemann, S. G., Flaherty, A. W., Eskandar, E. N., Kostyk, S. K., et al. (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)70039-4PMID21419704
Kaplitt MG, Feigin A, Tang C, et al. (June 2007). “Safety and tolerability of gene therapy with an adeno-associated virus (AAV) borne GAD gene for Parkinson's disease: an open label, phase I trial”. Lancet369 (9579): 2097?105. doi:10.1016/S0140-6736(07)60982-9. PMID17586305.
Erles K, Rohde V, Thaele M, Roth S, Edler L, Schlehofer JR (November 2001). “DNA of adeno-associated virus (AAV) in testicular tissue and in abnormal semen samples”. Human Reproduction16 (11): 2333–7. doi:10.1093/humrep/16.11.2333. PMID11679515.
Bohenzky RA, LeFebvre RB, Berns KI (October 1988). “Sequence and symmetry requirements within the internal palindromic sequences of the adeno-associated virus terminal repeat”. Virology166 (2): 316–27. doi:10.1016/0042-6822(88)90502-8. PMID2845646.
Wang XS, Ponnazhagan S, Srivastava A (July 1995). “Rescue and replication signals of the adeno-associated virus 2 genome”. Journal of Molecular Biology250 (5): 573–80. doi:10.1006/jmbi.1995.0398. PMID7623375.
Im DS, Muzyczka N (May 1990). “The AAV origin binding protein Rep68 is an ATP-dependent site-specific endonuclease with DNA helicase activity”. Cell61 (3): 447–57. doi:10.1016/0092-8674(90)90526-K. PMID2159383.
Cassinotti P, Weitz M, Tratschin JD (November 1988). “Organization of the adeno-associated virus (AAV) capsid gene: mapping of a minor spliced mRNA coding for virus capsid protein 1”. Virology167 (1): 176–84. doi:10.1016/0042-6822(88)90067-0. PMID2847413.
Rabinowitz JE, Samulski RJ (December 2000). “Building a better vector: the manipulation of AAV virions”. Virology278 (2): 301–8. doi:10.1006/viro.2000.0707. PMID11118354.
Mori S, Wang L, Takeuchi T, Kanda T (December 2004). “Two novel adeno-associated viruses from cynomolgus monkey: pseudotyping characterization of capsid protein”. Virology330 (2): 375–83. doi:10.1016/j.virol.2004.10.012. PMID15567432.
Bartlett JS, Samulski RJ, McCown TJ (May 1998). “Selective and rapid uptake of adeno-associated virus type 2 in brain”. Human Gene Therapy9 (8): 1181–6. doi:10.1089/hum.1998.9.8-1181. PMID9625257.
Fischer AC, Beck SE, Smith CI, et al. (December 2003). “Successful transgene expression with serial doses of aerosolized rAAV2 vectors in rhesus macaques”. Molecular Therapy8 (6): 918–26. doi:10.1016/j.ymthe.2003.08.015. PMID14664794.
Nicklin SA, Buening H, Dishart KL, et al. (September 2001). “Efficient and selective AAV2-mediated gene transfer directed to human vascular endothelial cells”. Molecular Therapy4 (3): 174–81. doi:10.1006/mthe.2001.0424. PMID11545607.
Rabinowitz JE, Xiao W, Samulski RJ (December 1999). “Insertional mutagenesis of AAV2 capsid and the production of recombinant virus”. Virology265 (2): 274–85. doi:10.1006/viro.1999.0045. PMID10600599.
Shi W, Bartlett JS (April 2003). “RGD inclusion in VP3 provides adeno-associated virus type 2 (AAV2)-based vectors with a heparan sulfate-independent cell entry mechanism”. Molecular Therapy7 (4): 515–25. doi:10.1016/S1525-0016(03)00042-X. PMID12727115.
Manno CS, Chew AJ, Hutchison S, et al. (April 2003). “AAV-mediated factor IX gene transfer to skeletal muscle in patients with severe hemophilia B”. Blood101 (8): 2963–72. doi:10.1182/blood-2002-10-3296. PMID12515715.
Qing K, Mah C, Hansen J, Zhou S, Dwarki V, Srivastava A (January 1999). “Human fibroblast growth factor receptor 1 is a co-receptor for infection by adeno-associated virus 2”. Nature Medicine5 (1): 71–7. doi:10.1038/4758. PMID9883842.
Summerford C, Bartlett JS, Samulski RJ (January 1999). “AlphaVbeta5 integrin: a co-receptor for adeno-associated virus type 2 infection”. Nature Medicine5 (1): 78–82. doi:10.1038/4768. PMID9883843.
Qiu J, Handa A, Kirby M, Brown KE (March 2000). “The interaction of heparin sulfate and adeno-associated virus 2”. Virology269 (1): 137–47. doi:10.1006/viro.2000.0205. PMID10725206.
Di Pasquale G, Davidson BL, Stein CS, et al. (October 2003). “Identification of PDGFR as a receptor for AAV-5 transduction”. Nature Medicine9 (10): 1306–12. doi:10.1038/nm929. PMID14502277.
Zaiss AK, Muruve DA (June 2005). “Immune responses to adeno-associated virus vectors”. Current Gene Therapy5 (3): 323–31. doi:10.2174/1566523054065039. PMID15975009.
Manno CS, Pierce GF, Arruda VR, et al. (March 2006). “Successful transduction of liver in hemophilia by AAV-Factor IX and limitations imposed by the host immune response”. Nature Medicine12 (3): 342–7. doi:10.1038/nm1358. PMID16474400.
Sabatino DE, Mingozzi F, Hui DJ, et al. (December 2005). “Identification of mouse AAV capsid-specific CD8+ T cell epitopes”. Molecular Therapy12 (6): 1023–33. doi:10.1016/j.ymthe.2005.09.009. PMID16263332.
Matsushita T, Elliger S, Elliger C, et al. (July 1998). “Adeno-associated virus vectors can be efficiently produced without helper virus”. Gene Therapy5 (7): 938–45. doi:10.1038/sj.gt.3300680. PMID9813665.
Chirmule N, Propert K, Magosin S, Qian Y, Qian R, Wilson J (September 1999). “Immune responses to adenovirus and adeno-associated virus in humans”. Gene Therapy6 (9): 1574–83. doi:10.1038/sj.gt.3300994. PMID10490767.
Ponnazhagan S, Mukherjee P, Yoder MC, et al. (April 1997). “Adeno-associated virus 2-mediated gene transfer in vivo: organ-tropism and expression of transduced sequences in mice”. Gene190 (1): 203–10. doi:10.1016/S0378-1119(96)00576-8. PMID9185868.
McCarty, D M; Monahan, P E; Samulski, R J (2001). “Self-complementary recombinant adeno-associated virus (scAAV) vectors promote efficient transduction independently of DNA synthesis”. Gene Therapy8 (16): 1248–54. doi:10.1038/sj.gt.3301514. PMID11509958.
Bainbridge, J. W. B., Smith, A. J., Barker, S. S., Robbie, S., Henderson, R., Balaggan, K., Viswanathan, A., et al. (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/NEJMoa0802268PMID18441371
Hauswirth, W. W., Aleman, T. S., Kaushal, S., Cideciyan, A. V., Schwartz, S. B., Wang, L., Conlon, T. J., et al. (2008). Treatment of Leber Congenital Amaurosis Due to RPE65Mutations by Ocular Subretinal Injection of Adeno-Associated Virus Gene Vector: Short-Term Results of a Phase I Trial. Human gene therapy, 19(10), 979–990. doi:10.1089/hum.2008.107PMID18774912
LeWitt, P. A., Rezai, A. R., Leehey, M. A., Ojemann, S. G., Flaherty, A. W., Eskandar, E. N., Kostyk, S. K., et al. (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)70039-4PMID21419704
Kaplitt MG, Feigin A, Tang C, et al. (June 2007). “Safety and tolerability of gene therapy with an adeno-associated virus (AAV) borne GAD gene for Parkinson's disease: an open label, phase I trial”. Lancet369 (9579): 2097?105. doi:10.1016/S0140-6736(07)60982-9. PMID17586305.
Erles K, Rohde V, Thaele M, Roth S, Edler L, Schlehofer JR (November 2001). “DNA of adeno-associated virus (AAV) in testicular tissue and in abnormal semen samples”. Human Reproduction16 (11): 2333–7. doi:10.1093/humrep/16.11.2333. PMID11679515.
Nault JC, Datta S, Imbeaud S, Franconi A, Mallet M, Couchy G, Letouzé E, Pilati C, Verret B, Blanc JF, Balabaud C, Calderaro J, Laurent A, Letexier M, Bioulac-Sage P, Calvo F, Zucman-Rossi J. "Recurrent AAV2-related insertional mutagenesis in human hepatocellular carcinomas" Nat Genet. 2015 Oct;47(10):1187-93. doi:10.1038/ng.3389. Epub 2015 Aug 24. PMID26301494
Bohenzky RA, LeFebvre RB, Berns KI (October 1988). “Sequence and symmetry requirements within the internal palindromic sequences of the adeno-associated virus terminal repeat”. Virology166 (2): 316–27. doi:10.1016/0042-6822(88)90502-8. PMID2845646.
Wang XS, Ponnazhagan S, Srivastava A (July 1995). “Rescue and replication signals of the adeno-associated virus 2 genome”. Journal of Molecular Biology250 (5): 573–80. doi:10.1006/jmbi.1995.0398. PMID7623375.
Im DS, Muzyczka N (May 1990). “The AAV origin binding protein Rep68 is an ATP-dependent site-specific endonuclease with DNA helicase activity”. Cell61 (3): 447–57. doi:10.1016/0092-8674(90)90526-K. PMID2159383.
Cassinotti P, Weitz M, Tratschin JD (November 1988). “Organization of the adeno-associated virus (AAV) capsid gene: mapping of a minor spliced mRNA coding for virus capsid protein 1”. Virology167 (1): 176–84. doi:10.1016/0042-6822(88)90067-0. PMID2847413.
Rabinowitz JE, Samulski RJ (December 2000). “Building a better vector: the manipulation of AAV virions”. Virology278 (2): 301–8. doi:10.1006/viro.2000.0707. PMID11118354.
Mori S, Wang L, Takeuchi T, Kanda T (December 2004). “Two novel adeno-associated viruses from cynomolgus monkey: pseudotyping characterization of capsid protein”. Virology330 (2): 375–83. doi:10.1016/j.virol.2004.10.012. PMID15567432.
Bartlett JS, Samulski RJ, McCown TJ (May 1998). “Selective and rapid uptake of adeno-associated virus type 2 in brain”. Human Gene Therapy9 (8): 1181–6. doi:10.1089/hum.1998.9.8-1181. PMID9625257.
Fischer AC, Beck SE, Smith CI, et al. (December 2003). “Successful transgene expression with serial doses of aerosolized rAAV2 vectors in rhesus macaques”. Molecular Therapy8 (6): 918–26. doi:10.1016/j.ymthe.2003.08.015. PMID14664794.
Nicklin SA, Buening H, Dishart KL, et al. (September 2001). “Efficient and selective AAV2-mediated gene transfer directed to human vascular endothelial cells”. Molecular Therapy4 (3): 174–81. doi:10.1006/mthe.2001.0424. PMID11545607.
Rabinowitz JE, Xiao W, Samulski RJ (December 1999). “Insertional mutagenesis of AAV2 capsid and the production of recombinant virus”. Virology265 (2): 274–85. doi:10.1006/viro.1999.0045. PMID10600599.
Shi W, Bartlett JS (April 2003). “RGD inclusion in VP3 provides adeno-associated virus type 2 (AAV2)-based vectors with a heparan sulfate-independent cell entry mechanism”. Molecular Therapy7 (4): 515–25. doi:10.1016/S1525-0016(03)00042-X. PMID12727115.
Manno CS, Chew AJ, Hutchison S, et al. (April 2003). “AAV-mediated factor IX gene transfer to skeletal muscle in patients with severe hemophilia B”. Blood101 (8): 2963–72. doi:10.1182/blood-2002-10-3296. PMID12515715.
Richter M, Iwata A, Nyhuis J, et al. (April 2000). “Adeno-associated virus vector transduction of vascular smooth muscle cells in vivo”. Physiological Genomics2 (3): 117–27. PMID11015590.
Summerford C, Bartlett JS, Samulski RJ (January 1999). “AlphaVbeta5 integrin: a co-receptor for adeno-associated virus type 2 infection”. Nature Medicine5 (1): 78–82. doi:10.1038/4768. PMID9883843.
Qiu J, Handa A, Kirby M, Brown KE (March 2000). “The interaction of heparin sulfate and adeno-associated virus 2”. Virology269 (1): 137–47. doi:10.1006/viro.2000.0205. PMID10725206.
Di Pasquale G, Davidson BL, Stein CS, et al. (October 2003). “Identification of PDGFR as a receptor for AAV-5 transduction”. Nature Medicine9 (10): 1306–12. doi:10.1038/nm929. PMID14502277.
Zaiss AK, Muruve DA (June 2005). “Immune responses to adeno-associated virus vectors”. Current Gene Therapy5 (3): 323–31. doi:10.2174/1566523054065039. PMID15975009.
Manno CS, Pierce GF, Arruda VR, et al. (March 2006). “Successful transduction of liver in hemophilia by AAV-Factor IX and limitations imposed by the host immune response”. Nature Medicine12 (3): 342–7. doi:10.1038/nm1358. PMID16474400.
Sabatino DE, Mingozzi F, Hui DJ, et al. (December 2005). “Identification of mouse AAV capsid-specific CD8+ T cell epitopes”. Molecular Therapy12 (6): 1023–33. doi:10.1016/j.ymthe.2005.09.009. PMID16263332.
Matsushita T, Elliger S, Elliger C, et al. (July 1998). “Adeno-associated virus vectors can be efficiently produced without helper virus”. Gene Therapy5 (7): 938–45. doi:10.1038/sj.gt.3300680. PMID9813665.