Article
TALEN-based gene correction for epidermolysis bullosa.
Molecular therapy : the journal of the American Society of Gene Therapy - 1 Jun 2013
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
Abstract excerpt
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 explored the possibility of using engineered transcription activator-like effector nucleases (TALEN) for precise genome...
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