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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...
Αποθηκεύτηκε σε:
| Τόπος έκδοσης: | Mol Ther |
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| Κύριοι συγγραφείς: | , , , , , , , , , , , |
| Μορφή: | Artigo |
| Γλώσσα: | Inglês |
| Έκδοση: |
American Society of Gene & Cell Therapy
2017
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| Θέματα: | |
| Διαθέσιμο Online: | 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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