Template-assisted sequence knock-in rescues skeletal and cardiac muscle function in a deletion model of Duchenne muscular dystrophy
Mol Ther. 2025 May 7:S1525-0016(25)00375-2. doi: 10.1016/j.ymthe.2025.05.005. Online ahead of print. ABSTRACT Duchenne muscular dystrophy (DMD) poses challenges in therapy design due to dystrophin’s complex …