Fig. 1.
The development history of AAV as a delivery tool for ocular gene therapy. The blue box represents the stage where humans have gradually explored the biological characteristics of AAV through cloning and sequencing since its discovery. The yellow box represents the stage in which humans attempt to stably express genes in animals and humans by continuously optimizing rAAV, and, for the first time, the effectiveness of AAV-based gene therapy has been verified in LCA patients, demonstrating the enormous potential of AAV-based gene therapy in the application of ocular diseases. The red box represents the stage of AAV-based gene therapy in the bench-to-bed stage of ocular diseases. At this stage, the efficacy and safety of AAV-based gene therapy have been further improved by screening promoters and AAV capsids specifically expressed in eye tissue and developing various viral delivery pathways. The increasing number of clinical trials targeting various eye diseases has also accumulated data for the application of AAV-based gene therapy in eye diseases.