New centers boost muscular dystrophy research

The National Institute of Arthritis and Musculoskeletal and Skin Diseases (NIAMS), the National Institute of Neurological Disorders and Stroke (NINDS) and the National Institute of Child Health and Human Development (NICHD), parts of the National Institutes of Health (NIH), have funded three new cooperative research centers for the muscular dystrophies (MD), a group of genetic diseases that result in muscle weakness and wasting. The three institutes will fund the centers at up to $1 million in direct costs per center per year for 5 years. The NIH expects to fund up to two additional centers in future years.

The centers, principal investigators, funding agencies and planned research include:

  • The University of Pittsburgh, Joseph C. Glorioso, Ph.D. (NIAMS). Scientists here will study gene and stem cell therapies to treat muscle disease, in particular Duchenne muscular dystrophy. One project will attempt to deliver and engraft muscle stem cells into diseased heart tissue without causing an immune response. Researchers will also use herpesvirus vectors in functional genomics studies to discover and characterize factors that guide stem cell maturation into muscle. In collaboration with scientists at the University of Missouri and Ohio State University, adeno-associated viral vectors will be exploited in preclinical studies in a dog MD model seeking the most effective ways to deliver gene therapy. Clinical disease outcomes will be carefully defined in patients with Duchenne and limb-girdle muscular dystrophies for the preparation of phase I gene therapy safety trials.
  • The University of Washington, Seattle, Jeffrey S. Chamberlain, Ph.D. (NICHD). This center will conduct studies to develop new gene therapies for Duchenne muscular dystrophy. Researchers have already achieved some success with viral vectors in delivering genes to mouse muscle. Investigators will use animal models to study several aspects of gene therapy to guid

Contact: Ray Fleming
NIH/National Institute of Arthritis and Musculoskeletal and Skin Diseases

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