Non-uniform dystrophin re-expression after CRISPR-mediated exon excision in the dystrophin/utrophin double-knockout mouse model of DMD
Britt Hanson, Sofia Stenler, Nina Ahlskog, Katarzyna Chwalenia, Nenad Svrzikapa, Anna M. L. Coenen-Stass, Marc S. Weinberg, Matthew J. A. Wood, Thomas C. Roberts
This study uses CRISPR to excise exon 23 in dKO mice, achieving partial dystrophin restoration but with limited efficacy and challenges in DNA repair.
Molecular Therapy — Nucleic Acids · Oct 23, 2022Peer-reviewedMolecular Biology