$7.3 million NIH grant establishes Wellstone Muscular Dystrophy Research Center at the UI

ellstone Center at the UI because we already have expertise, infrastructure and resources focused on muscular dystrophy, and our investigators have a long-standing record of success in translational research on muscular dystrophies and in providing advanced diagnostic services."

Muscular dystrophies are a diverse group of genetic diseases that cause progressive muscle wasting and weakness in affected individuals. The various types of muscular dystrophy differ in severity, age of onset, specific muscles affected and the rate at which symptoms progress. More than one million Americans have muscular dystrophy or a related neuromuscular disorder.

Campbell will lead an investigation on whether improving muscle membrane maintenance and repair can provide a basis for new treatments of Duchenne muscular dystrophy. Campbell previously identified important roles for two proteins called LARGE and dysferlin in muscle maintenance and repair. If the new studies show that increasing levels of either or both of the proteins produce a therapeutic effect, the UI team will look for ways, including pharmacological strategies, to modify the activity or expression of the proteins.

A second project, led by Katherine Mathews, M.D, UI associate professor of pediatrics and neurology, will concentrate on limb-girdle muscular dystrophy 2I (LGMD 2I). This form of muscular dystrophy is caused by mutations in Fukutin-related protein. However, different mutations in this one protein lead to widely varying clinical symptoms. The UI researchers will study the disease and its consequences in LGMD 2I patients and also will develop mouse models to learn more about the disease process and test potential treatments.

The third project, led by Baoli Yang, M.D., Ph.D., UI assistant professor of obstetrics and gynecology, will explore the possibility of using embryonic stem cells to treat muscular dystrophies. The researchers will work with mouse embryonic stem cells to disc

Contact: Jennifer Brown
University of Iowa

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