• New gene therapy restores dystrophin protein in patients with Duchenne muscular dystrophy

    7 days ago - By Gene Therapy

    Posted on: 4 May 2021,
    UT Southwestern scientists successfully employed a new type of gene therapy to treat mice with Duchenne muscular dystrophy , uniquely utilizing CRISPR-Cas9-based tools to restore a large section of the dystrophin protein that is missing in many DMD patients. The approach, described online today in the journal Science Advances, could lead to a treatment for DMD and inform the treatment of other inherited diseases.
    {loadposition user9}
    Read more ...