The center at the University of Pennsylvania, co-directed by the university's H. Lee Sweeney, Ph.D., and Kathryn R. Wagner, M.D., Ph.D., of The Johns Hopkins School of Medicine, will explore new strategies for treating a variety of muscular dystrophies (MD). Two laboratory projects are focused on ways to increase muscle growth, and another on examining compounds that may be able to inhibit enzymes involved in breaking down muscle tissue. Clinical trials will determine the safety and feasibility of a potential drug treatment for Duchenne Muscular Dystrophy (DMD). The core facility--a muscle physiology lab--will analyze MD mouse models. Other sites involved with this center are the University of Florida, Gainesville, and the NINDS Intramural Research Program.
Center scientists at Washington, D.C.'s Children's National Medical Center, under the direction of Eric P. Hoffman, Ph.D., and Diana M. Escolar, M.D., will study biochemical pathways that contribute to DMD. A clinical project aims to identify genetic modifiers of the disease, and two laboratory studies focus on muscle cell damage and muscle growth and remodeling in mice. The center also has a bioinformatics and computing core, as well as a clinical core to help support the Cooperative International Neuromuscular Research Group, an existing consortium of MD clinical investigators. Collaborating with the center is the University of Padova in Italy.
The center at the University of Iowa, directed by the university's Kevin P. Campbell, Ph.D., and Steven A. Moore, M.D., Ph.D., will explore therapeutic strategies for different muscular dystrophies. One project focuses on muscle membrane maintenance and repair, and another on potential embryonic stem cell treatment in mice. A third study involves people with Fukutin Related Protein MD and the development of a mouse model for further investigation of this disease. The center cores here will serve as a national resource for human muscle biopsies, fibroblast cell cultures and embryonic stem cells for MD researchers, and will provide advanced services for diagnosing the different dystrophies.
These new centers join three others already funded by the National Institutes of Health and the Muscular Dystrophy Association at the University of Washington, the University of Pittsburgh and the University of Rochester. The centers spring from the Muscular Dystrophy Community Assistance, Research and Education (MD-CARE) Act passed by Congress in 2001. The centers work individually and collaboratively, and are guided by a steering committee that includes representatives from each center. Each has both basic and clinical research projects, and one or more core facilities to support them. Centers must also make core resources or services available to the national muscular dystrophy research community.
MD is characterized by progressive weakness and degeneration of the skeletal or voluntary muscles which control movement. Researchers at the Senator Paul D. Wellstone Centers study various forms of MD, including Duchenne/Becker Muscular Dystrophy, Myotonic Dystrophy, Facioscapulohumeral Dystrophy, Limb-Girdle Muscular Dystrophy, and others. MD can affect people of all ages. Although some forms first become apparent in infancy or childhood, others may not appear until middle age or later.
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