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find Author "Ren Chengda" 1 results
  • Gene therapy for hereditary retinal diseases: hopes and challenges

    Hereditary retinal diseases (IRD) are a type of blinding eye disease with high genetic heterogeneity. In recent years, the rapid development of gene therapy technology has provided new possibilities for the treatment of IRD. Among them, recombinant adeno-associated virus (rAAV) has become the most concerned gene delivery vector at present due to its low immunogenicity, long-term stable transgenic expression and good retinal targeting. Raav-based gene replacement therapies have demonstrated good safety and efficacy in multiple clinical trials. For instance, the Luxturna® therapy for RPE65 gene mutation-related retinopathy has been successfully applied in clinical practice. However, the packaging capacity limitation of rAAV makes it difficult to deliver larger pathogenic genes, such as USH2A or ABCA4, etc. In addition, some IRD require precise gene modification rather than simple gene supplementation. To overcome these limitations, researchers are exploring a variety of strategies, including the splitting and delivery of biadeno-associated viral vectors, CRISPR/Cas9 gene editing technology, and the development of novel engineered capsids. Future research should focus on optimizing the safety and delivery efficiency of gene editing tools, establishing a more complete preclinical evaluation system, and further enhancing the tissue specificity of vectors. The breakthroughs in these technologies will promote the application of gene therapy in a wider range of IRD.

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