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New gene therapy restores dystrophin protein in patients with Duchenne muscular dystrophy – News-Medical.Net
UT Southwestern scientists successfully employed a new type of gene therapy to treat mice with Duchenne muscular dystrophy (DMD), uniquely utilizing CRISPR-Cas9-based…
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…
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