Progress in Gene Therapy for Rhodopsin Autosomal Dominant Retinitis Pigmentosa

Adv Exp Med Biol. 2019:1185:113-118. doi: 10.1007/978-3-030-27378-1_19.

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.

Keywords: Autosomal dominant retinitis pigmentosa; Gene therapy; Knockdown and replacement; Rhodpsin.

Publication types

  • Review

MeSH terms

  • Animals
  • Gene Silencing
  • Genes, Dominant
  • Genetic Therapy*
  • Mutation
  • Retinitis Pigmentosa / classification
  • Retinitis Pigmentosa / therapy*
  • Rhodopsin / genetics*

Substances

  • Rhodopsin