Gene therapy proves effective as experimental treatment for muscular dystrophy

This exciting result demonstrates the promise of gene therapy in someday treating muscular dystrophy in humans as well.

• University of Washington researchers say they have used a harmless virus to transmit a missing muscle-strengthening gene to all muscles of a rat's body, reversing the muscle wasting that characterizes muscular dystrophy.
• Up to now, finding a method that would deliver the missing gene to the entire body has limited research into using gene therapy against muscular dystrophy, the researchers said in a statement.
• Duchenne muscular dystrophy is a genetic disorder that affects one of every 3,500 newborn males.
• Characterized by lack of dystrophin protein production, a victim's muscles eventually weaken to the point where he cannot survive.
• Dr. Jeffrey Chamberlain, professor of neurology at the school's Muscular Dystrophy Cooperative Research Center, said the technique delivered "therapeutic levels of dystrophin to every skeletal and cardiac muscle of an adult, dystrophic mouse.
• These muscles include the heart, the muscles used during breathing, and all the limb muscles."
• Chamberlain, whose research is published in the August edition of Nature Medicine, said he's seeking government approval to conduct limited trials in people, although such an experiment may be years away.