Que. researchers uncover gene therapy treatment
Last Updated: Thursday, April 15, 2010 | 7:57 PM ET
CBC News
Researchers at Quebec City's Laval University say they have made steps toward a treatment of a severe form of muscular dystrophy.
Duchenne muscular dystrophy is a genetic muscle-wasting disorder that occurs in about one in every 3,500 male births.
Children with the disease have trouble walking as early as preschool age, and nearly all of them lose the ability to walk between ages seven and 12.
Typically, they die in their 20s because of weakness in their heart and lung muscles.
While there is no known cure, Laval researchers in partnership with French genome engineering company Cellectis said they have found a way to repair the mutated gene responsible for the dystrophy.
Dr. Jacques P. Tremblay, a microbiologist at Laval who headed the team, said up until now research on its treatment has focused on growing donor cells, then transplanting them throughout several muscles in the body.
Process involves correcting mutated gene
That process is time consuming and at best can only help treat one person at a time, Tremblay told CBC News.
Tremblay said Laval's approach involves correcting the mutated gene, which is already in the cells of the patients using enzymes known as meganucleases.
Laval's findings were published Thursday in the international scientific journal Gene Therapy.
"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," Tremblay said in a statement.
"We're still two to three years away from this stage. Subsequent stages, including human trials, could take even longer."
The research is funded by a number of Canadian health organizations including the Canadian Institutes of Health Research, Muscular Dystrophy Canada and the Amyotrophic Lateral Sclerosis Society of Canada.
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