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RNAi-Mediated Gene Suppression in a GCAP1(L151F) Cone-Rod Dystrophy Mouse Model

Dominant mutations occurring in the high-affinity Ca(2+)-binding sites (EF-hands) of the GUCA1A gene encoding guanylate cyclase-activating protein 1 (GCAP1) cause slowly progressing cone-rod dystrophy (CORD) in a dozen families worldwide. We developed a nonallele-specific adeno-associated virus (AAV...

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Detalles Bibliográficos
Autores principales:	Jiang, Li, Li, Tansy Z., Boye, Shannon E., Hauswirth, William W., Frederick, Jeanne M., Baehr, Wolfgang
Formato:	Online Artículo Texto
Lenguaje:	English
Publicado:	Public Library of Science 2013
Materias:	Research Article
Acceso en línea:	https://www.ncbi.nlm.nih.gov/pmc/articles/PMC3589431/ https://www.ncbi.nlm.nih.gov/pubmed/23472098 http://dx.doi.org/10.1371/journal.pone.0057676

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