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Has retinal gene therapy come of age? From bench to bedside and back to bench

19 Jun 2019

Abstract excerpt

Retinal gene therapy has advanced considerably in the past three decades. Initial efforts have been devoted to comprehensively explore and optimize the transduction abilities of gene delivery vectors, define the appropriate intraocular administration routes and obtain evidence of efficacy in animal models of inherited retinal diseases (IRDs). Successful translation in clinical trials of the initial promising...

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PMID: 31238338
DOI: 10.1093/hmg/ddz130

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