The UW has been awarded a five-year, $7 million grant to study molecular and cellular therapies for Duchenne muscular dystrophy.
The National Institute of Neurological Disease and Stroke, one of the National Institutes of Health, awarded the program project grant to Stanley Froehner, professor and chair of the Department of Physiology and Biophysics. Froehner will be joined in the multidisciplinary project by Jeff Chamberlain, professor of neurology; Stephen Hauschka, professor of biochemistry; and Stephen Tapscott, professor of neurology and full member of the Fred Hutchinson Cancer Research Center.
The group will study animal models of Duchenne muscular dystrophy, a severe, common form of the muscle wasting disease, to better understand the pathology of the disease.
Froehner is looking into the function of dystrophin, because muscular dystrophy has been linked to a defect in the gene that produces dystrophin. Chamberlain and Hauschka are studying stem cells as a means to deliver gene therapies for the disease to muscle cells throughout the body. Hauschka also is working on synthetic proteins to replace dystrophin. Tapscott is examining the regulation of compensatory genes related to the disease.
Scientists believe that dystrophin provides strength to the membranes surrounding muscles, thereby protecting muscle fibers from tears during contractions and giving the fibers more structural integrity. A lack of dystrophin, as is found in muscular dystrophy, greatly weakens the muscle fibers.
Newer evidence suggests that dystrophin is also part of a signaling complex across the membranes surrounding muscles. Froehner's group are also searching for a possible replacement for the signaling function of dystrophin.
This past October, the NIH named UW one of three new cooperative centers in muscular dystrophy, yielding a five-year, $6.9 million federal grant and a matching $1.6 million grant from the Muscular Dystrophy Association. Chamberlain is leading that center, which will develop gene therapies for Duchenne muscular dystrophy, use animal models to help develop human trials for gene therapy, and conduct translational studies on other types of the disease to develop gene therapies.