Novel therapeutics for inherited retinal dystrophies (IRDs) have rapidly evolved since groundbreaking clinical trials for LCA due to RPE65 mutations led to the first FDA‐approved in vivo gene therapy. Since then, advancements in viral vectors have led to more efficient AAV transduction and developed other viral vectors for gene augmentation therapy of large gene targets. Furthermore, significant developments in gene editing and RNA modulation technologies have introduced novel capabilities for treatment of autosomal dominant diseases, intronic mutations, and/or large genes otherwise unable to be treated with current viral vectors. We highlight strategies currently being evaluated in gene therapy clinical trials and promising preclinical developments for IRDs.

中文翻译：

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视网膜基因治疗的新前景。

自从由于RPE65突变导致的LCA的开创性临床试验导致了首个FDA批准的体内基因治疗以来，用于遗传性视网膜营养不良（IRD）的新型疗法已经迅速发展。从那时起，病毒载体的发展已导致更有效的AAV转导，并开发了用于大型基因靶标基因增强疗法的其他病毒载体。此外，基因编辑和RNA调节技术的重大发展引入了用于治疗常染色体显性疾病，内含子突变和/或大基因的新功能，这些疾病否则无法用当前的病毒载体进行治疗。我们重点介绍了目前正在基因治疗临床试验中评估的策略以及有前途的IRD临床开发。