Progress in Gene Therapy for Rhodopsin Autosomal Dominant Retinitis Pigmentosa

视紫红质常染色体显性遗传性视网膜色素变性基因治疗的进展

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Abstract

This brief review summarizes the major proof-of-concept gene therapy studies for autosomal dominant retinitis pigmentosa (RP) caused by mutations in the rhodopsin gene (RHO-adRP) that have been conducted over the past 20 years in various animal models. We have listed in tabular form the various approaches, gene silencing reagents, gene delivery strategies, and salient results from these studies.

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