title: Promising and delivering gene therapies for vision loss.
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.
authors.affiliation: Schepens Eye Research Institute, Massachusetts Eye and Ear Infirmary, Department of Ophthalmology, Harvard Medical School, Harvard University, 20 Staniford Street, Boston, MA 02114, USA.
authors.forename: Livia S
authors.initials: LS
authors.lastname: Carvalho
authors.affiliation: Schepens 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.
authors.forename: Luk H
authors.initials: LH
authors.lastname: Vandenberghe
mesh_terms: D001766:Blindness / Q000628:therapy*; D002986:Clinical Trials as Topic; D000229:Dependovirus / Q000235:genetics; D018014:Gene Transfer Techniques; D015316:Genetic Therapy / Q000379:methods*; D006801:Humans; D012162:Retinal Degeneration / Q000628:therapy*
keywords: AAV; Clinical trial; Gene therapy; LCA; Lentiviral vector; Retina
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