Abstract:Gene therapy research for fundus diseases has made significant progress recently, offering new hope for refractory ocular conditions. This review summarizes the latest developments, evaluates challenges, and explores future trends. Gene therapies for inherited retinal diseases, including commercialized RPE65-LCA therapy, clinical advances in retinitis pigmentosa(RP)subtypes and Stargardt disease, and the “bilateral effect” in Leber hereditary optic neuropathy(LHON)therapy, were highlighted in this review. Gene therapy strategies for complex disorders such as age-related macular degeneration(ARMD)and diabetic retinopathy(DR)were discussed. Advances in gene delivery systems, including adeno-associated virus(AAV)vectors, non-viral vectors, and CRISPR/Cas9 applications, were also analyzed. Furthermore, major challenges, including safety, immunogenicity, long-term efficacy, and personalized approaches were also discussed. Research indicates fundus gene therapy is transitioning from proof-of-concept to clinical application. With continued innovation and experience, it promises to provide precise treatment options, significantly improving visual outcomes for patients.
