Recombinant AAV-mediated BEST1 transfer to the retinal pigment epithelium: analysis of serotype-dependent retinal effects.

Mutations in the BEST1 gene constitute an underlying cause of juvenile macular dystrophies, a group of retinal disorders commonly referred to as bestrophinopathies and usually diagnosed in early childhood or adolescence. The disease primarily affects macular and paramacular regions of the eye leadin...

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Bibliographic Details
Main Authors: Karina E Guziewicz, Barbara Zangerl, András M Komáromy, Simone Iwabe, Vincent A Chiodo, Sanford L Boye, William W Hauswirth, William A Beltran, Gustavo D Aguirre
Format: Article
Language:English
Published: Public Library of Science (PLoS) 2013-01-01
Series:PLoS ONE
Online Access:http://europepmc.org/articles/PMC3797066?pdf=render