Delivering Therapeutic Genes to Muscle

The world's first human gene therapy trials for a neuromuscular disease builds on the successes of earlier MDA-funded studies. This potential therapy could provide powerful benefits to patients with muscular dystrophy as well as to those with heart disease and other conditions.

Although the first trials address the safety of injecting a therapeutic gene into a single muscle, MDA researchers are also working to deliver healthy genes to many muscles simultaneously, dramatically increasing the potential effectiveness of the therapy.

1. Healthy genes for the muscle proteins that are defective in limb-girdle muscular dystrophy are placed in a modified virus "carrier." 2. MDA researchers inject billions of these modified viruses carrying therapeutic genes into a muscle in the foot.

3. Carriers deposit the needed genes in muscle cells. Cells "read" the genes to create the missing muscle proteins.

If this approach effectively restores the missing muscle protein, additional trials by MDA investigators will test its potential to enhance muscle strength.