COLUMBIA, Mo. Approximately 250,000 people in the United States have some form of muscular dystrophy. Duchenne muscular dystrophy (DMD) is the most common type of the disease, predominantly affecting males. Boys with DMD will lose the ability to walk by their teens and typically die before the age of 30. For years, scientists have studied the use of gene therapy as a possible way to correct the muscle deterioration, but hurdles such as the need to treat all muscles in the body, including both skeletal muscle and heart muscle, have challenged researchers looking for an effective therapy until now.
In recent studies, published in Molecular Therapy and Human Gene Therapy, a team of University of Missouri researchers, led by Dongsheng Duan, associate professor of molecular microbiology and immunology, has found not only a delivery method that can reach every muscle of the body in a large animal model, but a therapy that will work on both skeletal muscle, the type found in arms and legs, and cardiac muscle, such as the heart.
"The difficult challenge with treating Duchenne muscular dystrophy, and other types of muscle-related diseases, is that the therapy must reach almost every muscle throughout the body," Duan said. "We have found that our new therapy, which uses a particular virus to deliver the gene therapy, reaches all of the muscles in large animals. This development raises the hope of whole body correction of Duchenne muscular dystrophy."
Patients with Duchnne muscular dystrophy have a gene mutation that disrupts the production of a protein known as dystrophin. Absence of this protein starts a chain reaction that eventually leads to muscle cell degeneration and death. Eventually, the damaged muscle tissue is replaced by fibrous, bony or fatty tissue and loses function. In the heart, this leads to severe heart disease and can place severe limitations on individuals afflicted with the disease.
In gene thera
|Contact: Christian Basi|
University of Missouri-Columbia