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Recombinant adeno-associated virus type 2-mediated gene delivery into the Rpe65(-/- )knockout mouse eye results in limited rescue

BACKGROUND: Leber's congenital amaurosis (LCA) is a severe form of retinal dystrophy. Mutations in the RPE65 gene, which is abundantly expressed in retinal pigment epithelial (RPE) cells, account for approximately 10–15% of LCA cases. In this study we used the high turnover, and rapid breeding...

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Detalles Bibliográficos
Autores principales: Lai, Chooi-May, Yu, Meaghan JT, Brankov, Meliha, Barnett, Nigel L, Zhou, Xiaohuai, Redmond, T Michael, Narfstrom, Kristina, Rakoczy, P Elizabeth
Formato: Texto
Lenguaje:English
Publicado: BioMed Central 2004
Materias:
Acceso en línea:https://www.ncbi.nlm.nih.gov/pmc/articles/PMC416492/
https://www.ncbi.nlm.nih.gov/pubmed/15109394
http://dx.doi.org/10.1186/1479-0556-2-3