Promising and delivering gene therapies for vision loss.
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ABSTRACT:
SUBMITTER: Carvalho LS
PROVIDER: S-EPMC4314511 | biostudies-literature | 2015 Jun
REPOSITORIES: biostudies-literature
Publications
Promising and delivering gene therapies for vision loss.
Carvalho Livia S LS Vandenberghe Luk H LH
Vision research 20140802 Pt B
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 po ...[more]
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