CRISPR therapy shows promise in early trial for fatal muscle disease

This early-phase study tested a new gene editing treatment called HG302 in 4 boys with Duchenne muscular dystrophy (DMD), a severe muscle-wasting disease. The therapy uses CRISPR technology to fix the genetic error and help muscles produce dystrophin, a protein essential for muscle strength. The main goal was to check safety, but researchers also looked for signs that the treatment restored dystrophin and improved muscle function.