Journal
VISION RESEARCH
Volume 111, Issue -, Pages 124-133Publisher
PERGAMON-ELSEVIER SCIENCE LTD
DOI: 10.1016/j.visres.2014.07.013
Keywords
Retina; Gene therapy; AAV; Lentiviral vector; LCA; Clinical trial
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Funding
- NIH [1DP1OD008267-01]
- Curing Kids Fund
- Foundation for Retina Research
- Foundation Fighting Blindness
- Research to Prevent Blindness
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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. (C) 2014 Elsevier Ltd. All rights reserved.
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