Article
COL7A1 Editing via CRISPR/Cas9 in Recessive Dystrophic Epidermolysis Bullosa.
Molecular therapy : the journal of the American Society of Gene Therapy - 1 Nov 2017
Hainzl Stefan, Peking Patricia, Kocher Thomas, Murauer Eva M, Larcher Fernando, Del Rio Marcela, Duarte Blanca, Steiner Markus, Klausegger Alfred, Bauer Johann W, Reichelt Julia, Koller Ulrich
Abstract excerpt
Designer nucleases allow specific and precise genomic modifications and represent versatile molecular tools for the correction of disease-associated mutations. In this study, we have exploited an ex vivo CRISPR/Cas9-mediated homology-directed repair approach for the correction of a frequent inherited mutation in exon 80 of COL7A1, which impairs type VII collagen expression, causing the severe blistering skin...
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