Abstract:Gene therapy research for retinal 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. We focus on gene therapy for inherited retinal diseases, including commercialized RPE65-LCA therapy, clinical advances in RP subtypes and Stargardt disease, and the "bilateral effect" in LHON therapy. We also discuss strategies for complex disorders like AMD and DR, and analyze advances in delivery systems including engineered AAV vectors, non-viral vectors, and CRISPR/Cas9 applications. The review addresses major challenges including safety, immunogenicity, long-term efficacy, and personalized approaches. Research indicates retinal 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.
