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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Autori principali: 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
Natura: Artigo
Lingua:Inglês
Pubblicazione: Nature Publishing Group 2013
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Original Article
Accesso online: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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