New Progress Against Muscular Dystrophy
August 30, 2018
Scientists at UT Southwestern’s O’Donnell Brain Institute have halted muscular dystrophy in the lab.
Dr. Eric Olson, Director of UT Southwestern’s Hamon Center for Regenerative Science and Medicine, leads the lab that made the breakthrough. His team used a technique called CRISPR to find a mutated gene responsible for Duchenne muscular dystrophy in laboratory animals, cut it out, and replace it with a healthy version of the gene.
Within weeks, all signs of Duchenne muscular dystrophy had disappeared.
“Children with DMD often die, either because their heart loses the strength to pump or their diaphragm becomes too weak to breathe,” Dr. Olson says. “This … would hopefully prevent that from happening.”
“Our strategy is different from other therapeutic approaches for DMD because it edits the mutation that causes the disease and restores normal expression,” adds Dr. Leonela Amoasii, lead author of the published study resulting from the work and Assistant Instructor of Molecular Biology in Dr. Olson’s lab. “But we have more to do before we can use this clinically.”