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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Autores principales: Guziewicz, Karina E., Zangerl, Barbara, Komáromy, András M., Iwabe, Simone, Chiodo, Vincent A., Boye, Sanford L., Hauswirth, William W., Beltran, William A., Aguirre, Gustavo D.
Formato: Artigo
Lenguaje:Inglês
Publicado: Public Library of Science 2013
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Research Article
Acceso en línea:https://ncbi.nlm.nih.gov/pmc/articles/PMC3797066/
https://ncbi.nlm.nih.gov/pubmed/24143172
https://ncbi.nlm.nih.govhttp://dx.doi.org/10.1371/journal.pone.0075666
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