Pharmacological and rAAV gene therapy rescue of visual functions in a blind mouse model of Leber congenital amaurosis.

Pharmacological and rAAV gene therapy rescue of visual functions in a blind mouse model of Leber congenital amaurosis.

Leber congenital amaurosis (LCA), a heterogeneous early-onset retinal dystrophy, accounts for approximately 15% of inherited congenital blindness. One cause of LCA is loss of the enzyme lecithin:retinol acyl transferase (LRAT), which is required for regeneration of the visual photopigment in the ret...

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Main Authors: Matthew L Batten, Yoshikazu Imanishi, Daniel C Tu, Thuy Doan, Li Zhu, Jijing Pang, Lyudmila Glushakova, Alexander R Moise, Wolfgang Baehr, Russell N Van Gelder, William W Hauswirth, Fred Rieke, Krzysztof Palczewski
Format: Article
Language:English
Published: Public Library of Science (PLoS) 2005-11-01
Series:PLoS Medicine
Online Access:http://europepmc.org/articles/PMC1274279?pdf=render

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http://europepmc.org/articles/PMC1274279?pdf=render

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