Efficient Cas9 nuclease-based editing in skeletal muscle via lipid nanoparticle delivery
Sukanya Iyer, Katelyn Daman, Yehui Sun, Amanda A. Tutto, Sarah E. Holbrook, Anya T. Joynt, Jing Yan, Prajakta Prasad Ambegaokar, Dongsheng Guo, Pengpeng Liu, Jennifer E. Stauffer, Stacy A. Maitland, Sang M. Lee, Thomas L. Gallagher, Gregory A. Cox, , Scot A. Wolfe
SORT LNPs enable efficient Cas9-mediated repair of a pathogenic allele in skeletal muscle in a mouse model of LGMDR7.
bioRxiv · May 12, 2025 · PreprintMolecular Biology