Bucher K, Rodriguez-Bocanegra E, Dauletbekov D, Fischer MD. Immune responses to retinal gene therapy using adeno-associated viral vectors - Implications for treatment success and safety. Prog Retin Eye Res. 2021;83:100915.

Article  CAS  PubMed  Google Scholar 

Kuzmin DA, Shutova MV, Johnston NR, Smith OP, Fedorin VV, Kukushkin YS, et al. The clinical landscape for AAV gene therapies. Nat Rev Drug Discov. 2021;20:173–4.

Article  CAS  PubMed  Google Scholar 

Wang D, Tai PWL, Gao G. Adeno-associated virus vector as a platform for gene therapy delivery. Nat Rev Drug Discov. 2019;18:358–78.

Article  CAS  PubMed  PubMed Central  Google Scholar 

Sarkar H, Moosajee M. Choroideremia: molecular mechanisms and therapies. Trends Mol Med. 2022;28:378–87.

Article  CAS  PubMed  Google Scholar 

Ail D, Malki H, Zin EA, Dalkara D. Adeno-associated virus (AAV) - based gene therapies for retinal diseases: where are we? Appl Clin Genet. 2023;16:111–30.

Article  CAS  PubMed  PubMed Central  Google Scholar 

Cideciyan AV, Jacobson SG, Beltran WA, Sumaroka A, Swider M, Iwabe S, et al. Human retinal gene therapy for Leber congenital amaurosis shows advancing retinal degeneration despite enduring visual improvement. Proc Natl Acad Sci USA. 2013;110:E517–25.

Article  CAS  PubMed  PubMed Central  Google Scholar 

Russell S, Bennett J, Wellman JA, Chung DC, Yu ZF, Tillman A, 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. Lancet. 2017;390:849–60.

Article  CAS  PubMed  PubMed Central  Google Scholar 

Smalley E. First AAV gene therapy poised for landmark approval. Nat Biotechnol. 2017;35:998–9.

Article  CAS  PubMed  Google Scholar 

Xiong W, Wu DM, Xue Y, Wang SK, Chung MJ, Ji X, et al. AAV cis-regulatory sequences are correlated with ocular toxicity. Proc Natl Acad Sci USA. 2019;116:5785–94.

Article  CAS  PubMed  PubMed Central  Google Scholar 

Chandler LC, McClements ME, Yusuf IH, Martinez-Fernandez de la Camara C, MacLaren RE, Xue K. Characterizing the cellular immune response to subretinal AAV gene therapy in the murine retina. Mol Ther Methods Clin Dev. 2021;22:52–65.

Article  CAS  PubMed  PubMed Central  Google Scholar 

Jin E, Yin H, Li X, Zhao M. Short-term outcomes after intravitreal injections of conbercept versus ranibizumab for the treatment of retinopathy of prematurity. Retina. 2018;38:1595–604.

Article  CAS  PubMed  Google Scholar 

Sun Z, Zhou H, Lin B, Jiao X, Luo Y, Zhang F, et al. Efficacy and safety of intravitreal conbercept injections in macular edema secondary to retinal vein occlusion. Retina. 2017;37:1723–30.

Article  CAS  PubMed  PubMed Central  Google Scholar 

Cheng SY, Luo Y, Malachi A, Ko J, Su Q, Xie J, et al. Low-dose recombinant adeno-associated virus-mediated inhibition of vascular endothelial growth factor can treat neovascular pathologies without inducing retinal vasculitis. Hum Gene Ther. 2021;32:649–66.

Article  CAS  PubMed  PubMed Central  Google Scholar 

Liu K, Song Y, Xu G, Ye J, Wu Z, Liu X, et al. Conbercept for treatment of neovascular age-related macular degeneration: results of the randomized phase 3 PHOENIX study. Am J Ophthalmol. 2019;197:156–67.

Article  CAS  PubMed  Google Scholar 

Timmers AM, Newmark JA, Turunen HT, Farivar T, Liu J, Song C, et al. Ocular inflammatory response to intravitreal injection of adeno-associated virus vector: relative contribution of genome and capsid. Hum Gene Ther. 2020;31:80–9.

Article  CAS  PubMed  Google Scholar 

Ramlogan-Steel CA, Murali A, Andrzejewski S, Dhungel B, Steel JC, Layton CJ. Gene therapy and the adeno-associated virus in the treatment of genetic and acquired ophthalmic diseases in humans: Trials, future directions and safety considerations. Clin Exp Ophthalmol. 2019;47:521–36.

Article  PubMed  Google Scholar 

Drag S, Dotiwala F, Upadhyay AK. Gene therapy for retinal degenerative diseases: progress, challenges, and future directions. Invest Ophthalmol Vis Sci. 2023;64:39.

Article  CAS  PubMed  PubMed Central  Google Scholar 

Dalkara D, Byrne LC, Klimczak RR, Visel M, Yin L, Merigan WH, et al. In vivo-directed evolution of a new adeno-associated virus for therapeutic outer retinal gene delivery from the vitreous. Sci Transl Med. 2013;5:189ra76.

Article  PubMed  Google Scholar 

Kern A, Schmidt K, Leder C, Muller OJ, Wobus CE, Bettinger K, et al. Identification of a heparin-binding motif on adeno-associated virus type 2 capsids. J Virol. 2003;77:11072–81.

Article  CAS  PubMed  PubMed Central  Google Scholar 

Isgrig K, McDougald DS, Zhu J, Wang HJ, Bennett J, Chien WW. AAV2.7m8 is a powerful viral vector for inner ear gene therapy. Nat Commun. 2019;10:427.

Article  CAS  PubMed  PubMed Central  Google Scholar 

Westhaus A, Cabanes-Creus M, Rybicki A, Baltazar G, Navarro RG, Zhu E, et al. High-throughput in vitro, ex vivo, and in vivo screen of adeno-associated virus vectors based on physical and functional transduction. Hum Gene Ther. 2020;31:575–89.

Article  CAS  PubMed  PubMed Central  Google Scholar 

Ramachandran PS, Lee V, Wei Z, Song JY, Casal G, Cronin T, et al. Evaluation of dose and safety of AAV7m8 and AAV8BP2 in the non-human primate retina. Hum Gene Ther. 2017;28:154–67.

Article  CAS  PubMed  PubMed Central  Google Scholar 

Grishanin R, Vuillemenot B, Sharma P, Keravala A, Greengard J, Gelfman C, et al. Preclinical evaluation of ADVM-022, a novel gene therapy approach to treating wet age-related macular degeneration. Mol Ther. 2019;27:118–29.

Article  CAS  PubMed  Google Scholar 

Kiss S, Grishanin R, Nguyen A, Rosario R, Greengard JS, Nieves J, et al. Analysis of aflibercept expression in NHPs following intravitreal administration of ADVM-022, a potential gene therapy for nAMD. Mol Ther Methods Clin Dev. 2020;18:345–53.

Article  CAS  PubMed  PubMed Central  Google Scholar 

Khanani AM, Thomas MJ, Aziz AA, Weng CY, Danzig CJ, Yiu G, et al. Review of gene therapies for age-related macular degeneration. Eye. 2022;36:303–11.

Article  PubMed  PubMed Central  Google Scholar 

Heier JS, Kherani S, Desai S, Dugel P, Kaushal S, Cheng SH, et al. Intravitreous injection of AAV2-sFLT01 in patients with advanced neovascular age-related macular degeneration: a phase 1, open-label trial. Lancet. 2017;390:50–61.

Article  CAS  PubMed  Google Scholar 

Ye GJ, Budzynski E, Sonnentag P, Miller PE, Sharma AK, Ver Hoeve JN, et al. Safety and biodistribution evaluation in cynomolgus macaques of rAAV2tYF-CB-hRS1, a recombinant adeno-associated virus vector expressing retinoschisin. Hum Gene Ther Clin Dev. 2015;26:165–76.

Article  CAS  PubMed  PubMed Central  Google Scholar 

Reichel FF, Dauletbekov DL, Klein R, Peters T, Ochakovski GA, Seitz IP, et al. AAV8 can induce innate and adaptive immune response in the primate eye. Mol Ther. 2017;25:2648–60.

Article  CAS