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IMAGE: In a mouse model and in human heart muscle cells, researchers used gene editing to modify specific DNA sequences and restore dystrophin production in mutant dystrophin genes.
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Credit: UT Southwestern Medical Center
DALLAS - April 30, 2021 - 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. ....

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