Lentiviral gene transfer of RPE65 rescues survival and function of cones in a mouse model of Leber congenital amaurosis.

Lentiviral gene transfer of RPE65 rescues survival and function of cones in a mouse model of Leber congenital amaurosis.

<h4>Background</h4>RPE65 is specifically expressed in the retinal pigment epithelium and is essential for the recycling of 11-cis-retinal, the chromophore of rod and cone opsins. In humans, mutations in RPE65 lead to Leber congenital amaurosis or early-onset retinal dystrophy, a severe f...

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Bibliographic Details
Main Authors: Alexis-Pierre Bemelmans, Corinne Kostic, Sylvain V Crippa, William W Hauswirth, Janis Lem, Francis L Munier, Mathias W Seeliger, Andreas Wenzel, Yvan Arsenijevic
Format: Article
Language:English
Published: Public Library of Science (PLoS) 2006-10-01
Series:PLoS Medicine
Online Access:https://doi.org/10.1371/journal.pmed.0030347

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https://doi.org/10.1371/journal.pmed.0030347

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