Cut and Paste: Efficient Homology-Directed Repair of a Dominant Negative KRT14 Mutation via CRISPR/Cas9 Nickases

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 1...

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Vydáno v:Mol Ther
Hlavní autoři: 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
Médium: Artigo
Jazyk:Inglês
Vydáno: American Society of Gene & Cell Therapy 2017
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Original Article
On-line přístup:https://ncbi.nlm.nih.gov/pmc/articles/PMC5675592/
https://ncbi.nlm.nih.gov/pubmed/28888469
https://ncbi.nlm.nih.govhttp://dx.doi.org/10.1016/j.ymthe.2017.08.015
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