Article
Cut and Paste: Efficient Homology-Directed Repair of a Dominant Negative KRT14 Mutation via CRISPR/Cas9 Nickases.
Molecular therapy : the journal of the American Society of Gene Therapy - 1 Nov 2017
Kocher Thomas, Peking Patricia, Klausegger Alfred, Murauer Eva Maria, Hofbauer Josefina Piñón, Wally Verena, Lettner Thomas, Hainzl Stefan, Ablinger Michael, Bauer Johann Wolfgang, Reichelt Julia, Koller Ulrich
Abstract excerpt
With the ability to induce rapid and efficient repair of disease-causing mutations, CRISPR/Cas9 technology is ideally suited for gene therapy approaches for recessively and dominantly inherited monogenic disorders. In this study, we have corrected a causal hotspot mutation in exon 6 of the keratin 14 gene (KRT14) that results in generalized severe epidermolysis bullosa simplex (EBS-gen sev), using a...
Read the complete abstract on PubMedTopics
Share this publication in a Topic to start or enrich a Post.