Single-cut gene therapy mitigates Duchenne muscular dystrophy in an accelerated rhesus monkey model
April 14, 2025
Despite being known for more than 150 years, Duchenne muscular dystrophy (DMD) remains an untreatable disease affecting approximately 1 of every 3,500-5,000 males. Muscles in patients express no or inactive dystrophin, rendering them weak and less mobile.