CRISPR Halts Duchenne Muscular Dystrophy Progression in Dogs; Single-Cut Gene Editing Technique Restores Dystrophin Expression by Up to 92% of Normal

For the first time, scientists have used CRISPR gene editing to halt the progression of Duchenne muscular dystrophy (DMD) in a large mammal (dogs), according to a study by University of Texas (UT) Southwestern that provides a strong indication that a lifesaving treatment may be in the pipeline. The research, published online on August 30, 2018 in Science, documents unprecedented improvement in the muscle fibers of dogs with DMD - the most common fatal genetic disease in children, caused by a mutation that inhibits the production of dystrophin, a protein critical for muscle function. The article is titled “Gene Editing Restores Dystrophin Expression in a Canine Model Of Duchenne Muscular Dystrophy.” Researchers used a single-cut gene-editing technique to restore dystrophin in muscle and heart tissue by up to 92 percent of normal levels. Scientists have estimated a 15 percent threshold is needed to significantly help patients. "Children with DMD often die either because their heart loses the strength to pump, or their diaphragm becomes too weak to breathe," said Dr. Eric Olson, Director of UT Southwestern's Hamon Center for Regenerative Science and Medicine. "This encouraging level of dystrophin expression would hopefully prevent that from happening." DMD, which affects one in 5,000 boys, leads to muscle and heart failure, and premature death by the early 30s. Patients are forced into wheelchairs as their muscles degenerate and eventually onto respirators as their diaphragms weaken. No effective treatment exists, though scientists have known for decades that a defect in the dystrophin gene causes the condition. The Science study establishes the proof-of-concept for single-cut gene editing in dystrophic muscle and represents a major step toward a clinical trial. Already Dr.
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