Gene therapy and genome surgery in the retina
作者： James E. DiCarlo , Vinit B. Mahajan , Stephen H. Tsang
DOI: 10.1172/JCI120429
关键词:
摘要: Precision medicine seeks to treat disease with molecular specificity. Advances in genome sequence analysis, gene delivery, and genome surgery have allowed clinician-scientists to treat genetic conditions at the level of their pathology. As a result, progress in treating retinal disease using genetic tools has advanced tremendously over the past several decades. Breakthroughs in gene delivery vectors, both viral and nonviral, have allowed the delivery of genetic payloads in preclinical models of retinal disorders and have paved the way for …
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