Researchers Devise Improved Gene-Editing Process for Duchenne Muscular Dystrophy

Regenerative medicine researchers at the University of Texas (UT) Southwestern Medical Center have developed an improved and simplified gene-editing technique using CRISPR/Cas9 tools to correct a common mutation that causes Duchenne muscular dystrophy. When researchers used the new single-cut technique on a new mouse model, which they also developed to better study the disease, the mice showed improved muscle quality and strength, the scientists report in Science Translational Medicine. The article is titled “Single-Cut Genome Editing Restores Dystrophin Expression in a New Mouse Model of Muscular Dystrophy.” “We think these advancements will be valuable for the field and can help us move closer to tackling this disease in humans,” said Dr. Eric Olson (photo), Director of the Hamon Center for Regenerative Science and Medicine and Co-Director of the Wellstone Muscular Dystrophy Cooperative Research Center at UT Southwestern. The new approach restored up to 90 percent of dystrophin protein expression throughout the skeletal muscles and the heart in the mouse model. The lack of dystrophin protein is what leads to muscle and heart failure, and eventually premature death, from Duchenne muscular dystrophy (DMD). UT Southwestern researchers are now using the improved technique in human DMD cells and expect they will ultimately be able to correct between 60 and 80 percent of human DMD mutations, said Dr. Olson, also Chairman of Molecular Biology at UT Southwestern. The newly created mouse model, which mimics a gene mutation commonly found in Duchenne muscular dystrophy patients, will be made available to others doing research in this area, said Dr. Olson. It can replace the commonly used version that is decades-old and unlike most of the DNA mutations that cause muscular dystrophy in humans.“We made a mouse model that more faithfully represents the human disease,” explained Dr. Olson.
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