Promising and delivering gene therapies for vision loss

Livia S Carvalho, Luk H Vandenberghe

Research output: Contribution to journalArticle

31 Citations (Scopus)

Abstract

The maturity in our understanding of the genetics and the pathogenesis of disease in degenerative retinal disorders has intersected in past years with a novel treatment paradigm in which a genetic intervention may lead to sustained therapeutic benefit, and in some cases even restoration of vision. Here, we review this prospect of retinal gene therapy, discuss the enabling technologies that have led to first-in-human demonstrations of efficacy and safety, and the road that led to this exciting point in time.

Original languageEnglish
Pages (from-to)124-133
Number of pages10
JournalVision Research
Volume111
Issue numberPart B
DOIs
Publication statusPublished - Jun 2015
Externally publishedYes

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