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$8 Million NIH Grant for Penn Medicine’s Wellstone Muscular Dystrophy Cooperative Research Center

October 5, 2010, Volume 57, No. 06


The National Institutes of Health (NIH) has renewed its  funding to the Paul D. Wellstone Muscular Dystrophy Cooperative Research  Center at the University  of Pennsylvania School of Medicine. The new grant totals over  $8 million for the next five years. The Center was established in 2005 to support muscular dystrophy research and is principally funded by the  National Institute of Arthritis and Musculoskeletal and Skin Diseases (NIAMS) and co-funded by the National Heart, Lung and Blood Institute.  (NHLBI).

The Penn School of Medicine is one of six Wellstone Centers in the  United States. Of the six original centers, the Penn Center was one of  three to be renewed, and is directed by Dr. H. Lee  Sweeney, chair of the department of physiology at Penn. All six Centers honor the memory of the late Senator Paul Wellstone, who  was a champion of muscular-dystrophy research and issues in Congress.

“This award continues to be the nucleus of a larger  translational-research initiative for muscular dystrophies at Penn,” said Dr. Sweeney.  Muscular Dystrophy (MD) is characterized by progressive weakness and  degeneration of the skeletal or voluntary muscles that control movement. Researchers at the Wellstone Centers study various forms of MD, including Duchenne/Becker Muscular Dystrophy, Myotonic Dystrophy,  Facioscapulohumeral Dystrophy, and Limb-Girdle Muscular  Dystrophy.

The Penn Center focuses on the molecular questions  behind the deterioration of muscle and its cellular scaffolding seen in MD. “What are the factors that lead to the failure of muscle to regenerate after repeated rounds of injury and repair?”asked Dr. Sweeney. “Why,  eventually, is muscle replaced with fat and scar tissue instead of being  repaired, leading to muscle wasting? What cells are the source of the fat  and fibrosis? Understanding what goes wrong in repair is necessary for the  success of future therapies, and in particular, eventual stem-cell  therapies.”

An important component of the Center is a collaborative project based  at the University of Florida to develop better MRI-based methodologies for  imaging fat and fibrosis in a variety of human muscular dystophies. This is critical for the evaluation of future therapeutic interventions in the muscular dystrophies.

With the new funds, the Penn Center will also start a training core focused on educating the next generation of scientists and physicians in  the research of muscle biology and MD. This core, co-directed by Dr. E. Michael Ostap, director of the Pennsylvania Muscle Institute (PMI) and professor of physiology, will partner with the PMI and  Penn’s existing NIAMS-sponsored training program to offer workshops and  symposia starting in spring of 2011.  “We are excited about the new educational opportunities provided by  this partnership for Penn Medicine, and also for the muscle community  at-large via web-based dissemination of our programs,” said Dr. Ostap.

Almanac - October 5, 2010, Volume 57, No. 06