Pharmacological and rAAV Gene Therapy Rescue of Visual Functions in a Blind Mouse Model of Leber Congenital Amaurosis

BACKGROUND: Leber congenital amaurosis (LCA), a heterogeneous early-onset retinal dystrophy, accounts for ~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 reti...

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Detalles Bibliográficos
Autores principales: Batten, Matthew L, Imanishi, Yoshikazu, Tu, Daniel C, Doan, Thuy, Zhu, Li, Pang, Jijing, Glushakova, Lyudmila, Moise, Alexander R, Baehr, Wolfgang, Van Gelder, Russell N., Hauswirth, William W, Rieke, Fred, Palczewski, Krzysztof
Formato: Texto
Lenguaje:English
Publicado: Public Library of Science 2005
Materias:
Research Article
Acceso en línea:https://www.ncbi.nlm.nih.gov/pmc/articles/PMC1274279/
https://www.ncbi.nlm.nih.gov/pubmed/16250670
http://dx.doi.org/10.1371/journal.pmed.0020333

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