Dystrophin is a major protein involved in the functionality of muscles. If a mutation occurs, the production of dystrophin becomes inhibited which leads to the development of Duchenne muscular dystrophy (DMD). DMD usually leads to muscle or heart failure, followed by premature death. There was no effective treatment available till date.

Now, for the first time, a research team from UT Southwestern has used a mechanism of CRISPR gene editing to stop the development of DMD. This technique can restore dystrophin levels in muscles up to 92%. This scientific finding was published in the journal, Science.

Dr. Eric Olson, Director of UT Southwestern's Hamon Center for Regenerative Science and Medicine, said "Children with DMD often die either because their heart loses the strength to pump, or their diaphragm becomes too weak to breathe. This encouraging level of dystrophin expression would hopefully prevent that from happening."

Dr. Leonela Amoasii, lead author of the study and Assistant Instructor of Molecular Biology in Dr. Olson's lab, said "Our strategy is different from other therapeutic approaches for DMD because it edits the mutation that causes the disease and restores normal expression of the repaired dystrophin. But we have more to do before we can use this clinically."

Next, the research team will proceed with the checking of dystrophin’s stability post gene editing and occurrence of side effects, if any. In the meantime, a biotech firm named Exonics Therapeutics Inc. has licensed the technology from UT Southwestern.