Duke Results Suggest CRISPR/Cas9-Enabled Gene Editing Might Be Used to Effectively Treat Over Half of Patients with Duchenne Muscular Dystrophy (DMD)

Duke researchers have demonstrated a genetic therapeutic technique that has the potential to treat more than half of the patients suffering from Duchenne muscular dystrophy (DMD). One of the challenges of treating DMD is that the disease can be caused by mutations in a number of different DNA sequences, and few of these mutations occur with any substantial frequency. The new technique, however, gets around this sticking point by targeting a large region of the (DMD) gene that contains many different mutations. The new study was published online on February 18, 2015 in Nature Communications. The article was entitled, “"Multiplex CRISPR/Cas9-Based Genome Editing for Correction of Dystrophin Mutations That Cause Duchenne Muscular Dystrophy." "There are no effective therapies currently available for people with DMD," said Dr. Charles Gersbach, Assistant Professor of Biomedical Engineering at Duke University. "DMD patients are in a wheelchair by age 10 and typically die in their 20s. They have nothing to stop this right now, and we're trying to work on that." DMD is caused by problems with the body's ability to produce dystrophin, a long-chain protein that binds the interior of a muscle fiber to its surrounding support structure. Dystrophin is coded by a gene with 79 genetic "chunks" called exons. If any one exon incurs a debilitating mutation, the dystrophin chain does not get built. Without dystrophin providing support, muscle tends to shred and slowly deteriorate. The disease affects one in 3,500 newborn males. The mutation is on the X chromosome so female children with two copies of X should have at least one functioning copy of the gene.
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