New gene therapy approach shows promise for Duchenne muscular dystrophy

Design and test of split intein constructs to assemble FL-dystrophin. Credit: Nature Communications (2024). DOI: 10.1038/s41467-024-50569-6 Indiana University School of Medicine researchers have made a significant breakthrough in developing a new gene therapy approach that restores full-length dystrophin protein, which could lead to new treatments for people with Duchenne muscular dystrophy (DMD). The study, published in Nature Communications, demonstrates the effectiveness of their novel gene therapy technology in improving muscle tissue and overall strength in mice models with DMD. DMD is a genetic disorder caused by mutations in the DMD gene, resulting in a lack of the protein dystrophin. This Read More