Natural history of cone disease in the murine model of Leber congenital amaurosis due to CEP290 mutation: determining the timing and expectation of therapy.

Natural history of cone disease in the murine model of Leber congenital amaurosis due to CEP290 mutation: determining the timing and expectation of therapy.

Mutations in the CEP290 (cilia-centrosomal protein 290 kDa) gene in Leber congenital amaurosis (LCA) cause early onset visual loss but retained cone photoreceptors in the fovea, which is the potential therapeutic target. A cone-only mouse model carrying a Cep290 gene mutation, rd16;Nrl-/-, was engin...

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Bibliographic Details
Main Authors: Shannon E Boye, Wei-Chieh Huang, Alejandro J Roman, Alexander Sumaroka, Sanford L Boye, Renee C Ryals, Melani B Olivares, Qing Ruan, Budd A Tucker, Edwin M Stone, Anand Swaroop, Artur V Cideciyan, William W Hauswirth, Samuel G Jacobson
Format: Article
Language:English
Published: Public Library of Science (PLoS) 2014-01-01
Series:PLoS ONE
Online Access:http://europepmc.org/articles/PMC3966841?pdf=render

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http://europepmc.org/articles/PMC3966841?pdf=render

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