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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Bibliographische Detailangaben
Hauptverfasser: 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
Format: Artigo
Sprache:Inglês
Veröffentlicht: Nature Publishing Group 2013
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Original Article
Online Zugang: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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