New CRISPR/Cas9 Gene-Editing Technique Abolishes Splice Sites for Frequently Mutated Exons in Duchenne Muscular Dystrophy (DMD) Heart Muscle Cells

Scientists have developed a CRISPR/Cas9 gene-editing technique that can potentially correct a majority of the 3,000 mutations that cause Duchenne muscular dystrophy (DMD) by making a single cut at strategic points along the patient’s DNA, according to a study from the University of Texas (UT) Southwestern Medical Center. The method, successfully tested in heart muscle cells from patients, offers an efficient alternative to the daunting task of developing an individualized molecular treatment for each gene mutation that causes DMD. It also opens up possible new treatment approaches for other diseases that have thus far required more intrusive methods to correct single-gene mutations. Scientists say the new strategy enhances the accuracy for surgical-like editing of the human genome, correcting mistakes in the DNA sequence that cause devastating diseases like DMD, a deadly condition caused by defects in the dystrophin gene. Normally, the dystrophin protein helps strengthen muscle fibers. “This is a significant step,” said Dr. Eric Olson, Director of UT Southwestern’s Hamon Center for Regenerative Science and Medicine. “We’re hopeful this technique will eventually alleviate pain and suffering, perhaps even save the lives, of DMD patients who have a wide range of mutations and, unfortunately, have had no other treatment options to eliminate the underlying cause of the disease.” A study published online on January 31, 2018 in Science Advances documents the success of the new CRISPR/Cas9 gene-editing technique designed to treat DMD. The open-access article is titled “Correction of Diverse Muscular Dystrophy Mutations in Human Engineered Heart Muscle by Single-Site Genome Editing.” In the article abstract, the authors, including Dr. Olson, state the following.
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