TALEN-based Gene Correction for Epidermolysis Bullosa

Recessive dystrophic epidermolysis bullosa (RDEB) is characterized by a functional deficit of type VII collagen protein due to gene defects in the type VII collagen gene (COL7A1). Gene augmentation therapies are promising, but run the risk of insertional mutagenesis. To abrogate this risk, we explor...

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Detalles Bibliográficos
Autores principales: Osborn, Mark J, Starker, Colby G, McElroy, Amber N, Webber, Beau R, Riddle, Megan J, Xia, Lily, DeFeo, Anthony P, Gabriel, Richard, Schmidt, Manfred, Von Kalle, Christof, Carlson, Daniel F, Maeder, Morgan L, Joung, J Keith, Wagner, John E, Voytas, Daniel F, Blazar, Bruce R, Tolar, Jakub
Formato: Artigo
Lenguaje:Inglês
Publicado: Nature Publishing Group 2013
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Original Article
Acceso en línea:https://ncbi.nlm.nih.gov/pmc/articles/PMC3677309/
https://ncbi.nlm.nih.gov/pubmed/23546300
https://ncbi.nlm.nih.govhttp://dx.doi.org/10.1038/mt.2013.56
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