Duchenne muscular dystrophy (DMD), caused by mutations in the dystrophin gene, is the most common, disabling and lethal muscle disease, afflicting one of every 3500 males. The progressive muscle wasting and weakness usually confine the patients to a wheelchair in their early teens, and lead to death by their early twenties due to respiratory or cardiac failure. Recently a few groups have reported that inhibition of myostatin, a TGF-?-like growth inhibitor of skeletal muscle, can promote growth of both normal and dystrophic muscles by increasing the muscle mass and the subsequent improvement in muscle total strength. In this proposal, we will use the DMD mouse (mdx) as the disease model, the non-pathogenic adeno-associated virus (AAV) as the gene transfer vehicle and myostatin propeptide as the therapeutic gene, to test the general hypothesis that gene therapy of DMD with myostatin inhibitors can improve muscle growth and ameliorate the pathologies of DMD in the mdx mouse model.
|Effective start/end date||9/1/06 → 2/28/08|
- Children's Hospital of Pittsburgh
Duchenne Muscular Dystrophy
Inbred mdx Mouse