Summary:
- This article discusses a new gene therapy approach that could potentially treat a rare genetic disorder called Duchenne muscular dystrophy (DMD).
- DMD is a debilitating disease that causes progressive muscle weakness and eventually leads to death, and currently there is no cure.
- The gene therapy involves using a modified virus to deliver a functional version of the dystrophin gene, which is missing or defective in people with DMD, to the muscles, potentially restoring muscle function and slowing the progression of the disease.