H. Büning, L. Perabo u. a.: Recent developments in adeno-associated virus vector technology. In: The journal of gene medicine. Band 10, Nummer 7, Juli 2008, S. 717–733. doi:10.1002/jgm.1205. PMID 18452237. (Review).
Stephen Russell et al.: Efficacy and safety of voretigene neparvovec (AAV2-hRPE65v2) in patients with RPE65-mediated inherited retinal dystrophy: a randomised, controlled, open-label, phase 3 trial. In: Lancet (London, England). Band390, Nr.10097, 26. August 2017, S.849–860, doi:10.1016/S0140-6736(17)31868-8, PMID 28712537, PMC 5726391 (freier Volltext).
M. Dominik Fischer et al.: Safety and Vision Outcomes of Subretinal Gene Therapy Targeting Cone Photoreceptors in Achromatopsia: A Nonrandomized Controlled Trial. In: JAMA Ophthalmology. 30. April 2020, doi:10.1001/jamaophthalmol.2020.1032, PMID 32352493, PMC 7193523 (freier Volltext).
M. Dominik Fischer et al.: Safety and Vision Outcomes of Subretinal Gene Therapy Targeting Cone Photoreceptors in Achromatopsia: A Nonrandomized Controlled Trial. In: JAMA Ophthalmology. 30. April 2020, doi:10.1001/jamaophthalmol.2020.1032, PMID 32352493, PMC 7193523 (freier Volltext).
Hui Wang, Yuxin Chen, Jun Lv, Xiaoting Cheng, Qi Cao, Daqi Wang, Longlong Zhang, Yilai Shu et al.: Bilateral gene therapy in children with autosomal recessive deafness 9: single-arm trial results. In: Nature Medicine, 5. Juni 2024; doi:10.1038/s41591-024-03023-5 (englisch).
H. Büning, L. Perabo u. a.: Recent developments in adeno-associated virus vector technology. In: The journal of gene medicine. Band 10, Nummer 7, Juli 2008, S. 717–733. doi:10.1002/jgm.1205. PMID 18452237. (Review).
D. R. Deyle, D. W. Russell: Adeno-associated virus vector integration. In: Current opinion in molecular therapeutics. Band 11, Nummer 4, August 2009, S. 442–447, PMID 19649989, PMC 2929125 (freier Volltext) (Review).
Stephen Russell et al.: Efficacy and safety of voretigene neparvovec (AAV2-hRPE65v2) in patients with RPE65-mediated inherited retinal dystrophy: a randomised, controlled, open-label, phase 3 trial. In: Lancet (London, England). Band390, Nr.10097, 26. August 2017, S.849–860, doi:10.1016/S0140-6736(17)31868-8, PMID 28712537, PMC 5726391 (freier Volltext).
M. Dominik Fischer et al.: Safety and Vision Outcomes of Subretinal Gene Therapy Targeting Cone Photoreceptors in Achromatopsia: A Nonrandomized Controlled Trial. In: JAMA Ophthalmology. 30. April 2020, doi:10.1001/jamaophthalmol.2020.1032, PMID 32352493, PMC 7193523 (freier Volltext).
M. Dominik Fischer et al.: Safety and Vision Outcomes of Subretinal Gene Therapy Targeting Cone Photoreceptors in Achromatopsia: A Nonrandomized Controlled Trial. In: JAMA Ophthalmology. 30. April 2020, doi:10.1001/jamaophthalmol.2020.1032, PMID 32352493, PMC 7193523 (freier Volltext).