Gene editing technique halts Duchenne muscular dystrophy in dogs
Gene editing technique halts Duchenne muscular dystrophy in dogs
Source: Xinhua| 2018-08-31 03:07:29|Editor: Liangyu
WASHINGTON, Aug. 30 (Xinhua) -- American scientists have for the first time used gene editing technology to halt the progression of Duchenne muscular dystrophy (DMD) in a large mammal, providing a potential lifesaving treatment for the most common fatal genetic disease in children.
The study published on Thursday in the journal Science reported the improvement in the muscle fibers of dogs with DMD, which is caused by a mutation that inhibits the production of dystrophin, a protein critical for muscle function.
Researchers from the University of Texas Southwestern Medical Center used a single-cut gene-editing technique to restore dystrophin in muscle and heart tissue by up to 92 percent of normal levels. The 15-percent threshold is needed to significantly help patients, according to scientists.
DMD, which affects one in 5,000 boys, leads to muscle and heart failure, and premature death by the early 30s. No effective treatment exists, though scientists have known for decades that a defect in the dystrophin gene causes the condition.
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