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

New gene therapy restores dystrophin protein in patients with Duchenne muscular dystrophy
UT Southwestern scientists successfully employed a new type of gene therapy to treat mice with Duchenne muscular dystrophy (DMD), 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.
Thousands of different mutations causing Duchenne have been identified, but they tend to cluster into certain parts of the dystrophin gene."
Eric Olson, Ph.D., Professor and Founding Chair, Molecular Biology, UT Southwestern

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