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Replacement Gene Therapy with a Human <i>RPGRIP1</i> Sequence Slows Photoreceptor Degeneration in a Murine Model of Leber Congenital Amaurosis

2010-04-12

Abstract excerpt

Genetic defect in RPGR-interacting protein 1 (RPGRIP1) is a known cause of Leber congenital amaurosis (LCA), a severe, inherited form of retinal degeneration. Here, Pawlyk et al. report results from studies evaluating the efficacy of AAV8-mediated replacement gene therapy in a mouse model of LCA carrying a targeted disruption of RPGRIP1. RPGR-interacting protein-1 (RPGRIP1) is localized in the photoreceptor-connec...

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Literature Corpus work
28cae1ba-27c8-5c6e-adc2-5079718c7364
DOI
10.1089/hum.2009.218
Open publication

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Replacement Gene Therapy with a Human <i>RPGRIP1</i> Sequence Slows Photoreceptor Degeneration in a Murine Model of Leber Congenital AmaurosisDOI 10.1089/hum.2009.218
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