A muscular dystrophy fix?
Scientists have used gene editing to correct the mutations behind a form of muscular dystrophy in dogs—a major breakthrough that raises hopes the same procedure could be used to cure the disease in humans. The most common fatal genetic condition in children, Duchenne muscular dystrophy, is caused by a genetic mutation that prevents the body from producing dystrophin, a protein essential for strong muscle fibers. If the gene is mutated, muscles—including the heart and diaphragm—waste away, reports The Guardian (U.K.). For this study, researchers used the gene-editing technology CRISPR to restore dystrophin production in four dogs. Within weeks of receiving the injection-administered treatment, the dogs had significantly improved levels of dystrophin: a 92 percent correction in the heart and a 58 percent change in the diaphragm. The researchers estimate that as little as 15 percent improvement could dramatically help people with Duchenne. They are now planning more extensive studies on dogs. “If everything were to continue smoothly,” says lead researcher Eric Olson, from the University of Texas Southwestern Medical Center, “we might be able to anticipate moving into a human trial in a few years.”