CEP290 gene transfer rescues Leber Congenital Amaurosis cellular phenotype

Mutations in CEP290 are the most common cause of Leber congenital amaurosis (LCA), a severe inherited retinal degenerative disease for which there is currently no cure. Autosomal recessive CEP290-associated LCA is a good candidate for gene-replacement therapy, and cells derived from affected individ...

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Auteurs principaux: Burnight, E.R., Wiley, L.A., Drack, A.V., Braun, T.A., Anfinson, K.R., Kaalberg, E.E., Halder, J.A., Affatigato, L.M., Mullins, R.F., Stone, E.M., Tucker, B.A.
Format: Artigo
Langue:Inglês
Publié: 2014
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Article
Accès en ligne:https://ncbi.nlm.nih.gov/pmc/articles/PMC4188442/
https://ncbi.nlm.nih.gov/pubmed/24807808
https://ncbi.nlm.nih.govhttp://dx.doi.org/10.1038/gt.2014.39
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