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Vision Res. 2015 Jun;111(Pt B):124-33. doi: 10.1016/j.visres.2014.07.013. Epub 2014 Aug 2.

Promising and delivering gene therapies for vision loss.

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  • 1Schepens Eye Research Institute, Massachusetts Eye and Ear Infirmary, Department of Ophthalmology, Harvard Medical School, Harvard University, 20 Staniford Street, Boston, MA 02114, USA.
  • 2Schepens Eye Research Institute, Massachusetts Eye and Ear Infirmary, Department of Ophthalmology, Harvard Medical School, Harvard University, 20 Staniford Street, Boston, MA 02114, USA. Electronic address: luk_vandenberghe@meei.harvard.edu.

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.

Copyright © 2014 Elsevier B.V. All rights reserved.

KEYWORDS:

AAV; Clinical trial; Gene therapy; LCA; Lentiviral vector; Retina

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