CRISPR gene editing in dogs stopped the progression of Duchenne muscular dystrophy, and scientists think that it can work on humans too.
A new research claims that dogs suffering from DMD, a genetic disorder characterized by progressive muscular degeneration, improved after a single-cut gene-editing procedure restored dystrophin, a protein used for movement, to the muscles and the heart.
DMD is also a problem in humans that leads to death.
Gene Editing As Treatment For DMD
DMD, one of the nine types of muscular dystrophy, is a rare disorder caused by a mutation. According to the Center for Disease Control or Prevention (CDC), only one in every 7,250 males aged 5 to 24 are diagnosed with the disorder.
Patients with DMD often experience weakness beginning in their pelvis and legs, motor skill issues, shortness of breath and fatigue, swelling of feet, and overall muscle deterioration.
There is currently no available cure for DMD.
However, a research recently published in Science journal sparks hope for children suffering from the genetic disorder.
"Children with DMD often die either because their heart loses the strength to pump, or their diaphragm becomes too weak to breathe," stated Eric Olson of UT Southwestern's Hamon Center for Regenerative Science and Medicine. "This encouraging level of dystrophin expression would hopefully prevent that from happening."
Gene Editing In Dogs
Using the CRISPR gene-editing tool, scientists were able to restore dystrophin in four dogs by making a small cut in the faulty DNA. Then the test subjects were injected with harmless viruses that edited the genome of the muscles and the heart.
The trial was done over several weeks.
According to the study, scientists were able to restore dystrophin in the muscle and the heart tissue by 92 percent. Patients need 15 percent or greater improvement.
Curing DMD In Humans
The treatment of DMD in dogs is a promising start to finding the cure to the genetical disorder. Dr. Olson and his team have also previously successfully corrected mutations in mice and human cells by making a single strategic cut in the DNA. However, this is the first time that the treatment was trialed in a larger mammal.
The scientists are hoping to move on to human trials, but first, more research is needed to be conducted to see if the dystrophin levels remain stable after the trial. They also need to check if the process will give patients adverse side effects.
