Researchers from Universite Laval’s Faculty of Medicine and the CHUQ Research Center claim to have found a way to repair a defective gene that causes Duchenne muscular dystrophy.
The team created enzymes (meganucleases) that are able to fix a protein called dystrophin. Mutations of that protein lead to the development of this disease.
Duchenne muscular dystrophy affects one in every 3,500 boys. It causes progressive muscle degeneration in childhood and most people die by the age of 25.
Dr. Jacques P. Tremblay is a microbiologist at Laval who headed the team. He told CBC News, "It must first be proven in laboratory animals that it is possible to insert a meganuclease targeting the dystrophin gene directly into muscle cells, and that this will induce the synthesis of dystrophin able to attach to the muscle fiber membrane. We’re still two to three years away from this stage. Subsequent stages, including human trials, could take even longer."
The findings were published in the international scientific journal Gene Therapy.