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

07/01/05

The University of Iowa has received a five-year, $7.3 million grant from the National Institute of Neurological Disorders and Stroke, part of the National Institutes of Health (NIH), to establish a Paul D. Wellstone Muscular Dystrophy Cooperative Research Center. The lead investigator is Kevin Campbell, Ph.D., the Roy J. Carver Chair of Physiology and Biophysics and department head in the UI Roy J. and Lucille A. Carver College of Medicine, and a Howard Hughes Medical Institute Investigator.

The UI center commemorates Sen. Paul Wellstone (D-Minn.) who died in 2002. Wellstone was a vocal supporter of research on Duchenne muscular dystrophy. In 2001, he introduced the Muscular Dystrophy Community Assistance, Research, and Education Amendments, signed into law in December 2001, mandating that the NIH establish centers of excellence for basic and clinical research into Duchenne and other forms of muscular dystrophy. After Wellstone's death, these centers were named in his honor. The center at the UI is the fourth such center to be funded by the NIH.

Campbell, who also is a UI professor of internal medicine and neurology, will be director of the UI center and Steven Moore, M.D., Ph.D., UI professor of pathology, will be co-director. Center researchers from clinical and basic science departments in the UI Carver College of Medicine and the UI College of Public Health will conduct three projects and run three core facilities. Studies and facilities will explore basic biological mechanisms that relate to possible treatments for muscular dystrophies, facilitate translational research on muscular dystrophies and provide advanced diagnostic services to patients and clinical trial participants.

"The Wellstone Centers promote basic, translational and clinical research on all forms of muscular dystrophy by providing resources and fostering collaboration by neuromuscular researchers nationwide," Campbell said. "We were successful in our bid to establish a Wellstone 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 discover the principles governing the differentiation of these cells into muscle-forming precursor cells that can be used for cell transplantation therapies in mouse models of muscular dystrophies.

Core facility A, directed by Campbell, is an administrative core, which will coordinate and oversee the center's work.

Core B, directed by Moore and Rita Barresi, Ph.D., UI research associate, is a muscle tissue/cell culture/diagnostics core that will serve as a national tissue and cell culture resource for muscular dystrophy studies, provide diagnostic expertise for patients, and examine muscle biopsies from clinical trial participants to evaluate new treatments.

Core C, directed by Yang and Roger Williamson, M.D., UI professor of obstetrics and gynecology, will alter muscular dystrophy genes in embryonic stem cells, and coax the gene-targeted embryonic stem cells to become muscle tissue. The ultimate goal is to provide cell-based models of disease for development of therapies against the diverse forms of muscular dystrophy. The core will be a national resource of targeted stem cells.

University of Iowa Health Care describes the partnership between the UI Roy J. and Lucille A. Carver College of Medicine and UI Hospitals and Clinics and the patient care, medical education and research programs and services they provide. Visit UI Health Care online at http://www.uihealthcare.com.

Source: Eurekalert & others

Last reviewed: By John M. Grohol, Psy.D. on 21 Feb 2009
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