Gene therapy shows promise for treating rare muscle diseaseost

A research team at the University of Alberta has made significant progress in developing a gene therapy to treat dysferlinopathy, a rare genetic condition that hampers the body's ability to repair damaged muscles. Dysferlinopathy results from mutations affecting the production of dysferlin, a protein essential for muscle repair. This condition causes progressive muscle weakness, typically appearing in late adolescence or early adulthood, and affects between 1 in 1,300 and 1 in 200,000 people. While not the most severe form of muscular dystrophy, it leads to lifelong disability, often requiring patients to use wheelchairs.