A team from UT Southwestern’s Hamon Center for Regenerative Science and Medicine used CRISPR gene-editing technique for the first time to halt the progression of Duchenne Muscular Dystrophy (DMD) in dogs. This breakthrough raises hopes in humans as thousands of children worldwide are suffering from DMD.
By using a single-cut gene-editing technique, the researchers managed to restore dystrophin in a dog’s muscles to up to 92 percent, which is a significant achievement given that the previous studies only got a 15% restoration.
What is Duchenne Muscular Dystrophy (DMD)?
DMD, as well as other muscular dystrophies, is a rare disease triggered by a genetic mutation. As the child is aging, Duchenne Muscular Dystrophy causes muscular weakness and immobility, while, in time, it makes day-by-day tasks almost impossible to perform.
While DMD affects one in 3,500 kids aged between 5 and 24, the disease is more prevalent in males. Girls with this specific genetic mutation will also show symptoms of DMD but much later and not to at the same intensity as boys.
CRISPR gene-editing-technique to tackle Duchenne Muscular Dystrophy (DMD)
“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,” explained Dr. Eric Olson from the UT Southwestern’s Hamon Center for Regenerative Science and Medicine.
The scientists managed to test how useful is the CRISPR gene-editing technique in tackling DMD in dogs. And, luckily, they were successful in restoring dystrophin in the muscles to up to 92% which means that the muscular dystrophy halted in the subjects of the experiment, dogs in this case.
“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,” said Dr. Leonela Amoasii, the study’s leading author.
Although these results are auspicious, the CRISPR gene-editing methods used on dogs are not yet clinically optimized for humans. Accordingly, there is much more work to do before the new therapy be used on human subjects with Duchenne Muscular Dystrophy (DMD).