In vivo gene disruption and homology-directed repair in muscles and muscle stem cells using CRISPR/Cas9
Bryan L. Peacker, Kuan‐Hung Lin, Amy Lam, Christopher L. Rios, Kexian Zhu, Jill Goldstein, Kathleen A. Messemer, Regan Ellis, Michael Florea, Heidi Kletzien, Naftali Horwitz, August D. Bratti, Umika S. Paul, Madeline Maier, K. C. Medha, , Amy J. Wagers
This study demonstrates CRISPR-HDR efficiency in postnatal cardiac, skeletal muscle, and muscle stem cells, showing developmental stage-dependent HDR rates and successful correction of a Dmd mutation in mdx mice.
bioRxiv · Jul 1, 2026 · PreprintCell Biology