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The Centers of Excellent program in muscular dystrophy research was established by the National Institutes of Health (NIH) in 2003, in honor of the late Senator Paul D. Wellstone of Minnesota. Established as part of the NIH enhancement and intensification of muscular dystrophy research associated with the MD-CARE Act, the centers are supported by five-year, renewable grants. Each of the six funded centers nationwide is comprised of research projects and core facilities with an emphasis on translational and clinical research for various muscular dystrophies. Each Center also has an administrative core and at least one one scientific resource core that serves as a resource for the national muscular dystrophy research effort. These Centers serve as focal points for research collaborations, communication, resource sharing and training of new muscular dystrophy researchers.
Collectively, the Wellstone MDCRCs are engaged in research on various forms of muscular dystrophy. Designed to accelerate progress toward effective treatments for muscular dystrophies through increased synergistic collaboration and coordination of research activities, they promote side-by-side basic, translational, and clinical research. Each center coordinates efforts to help bring together investigators at multiple sites.
We are pleased to announce that the University of Iowa's Paul D. Wellstone Muscular Dystrophy Cooperative Research Center (MDCRC), was recently renewed at full funding for years 11 through 15 (July 2015-June 2020). Center funding comes from the National Institute of Neurological Disorders and Stroke (NINDS), a subset of the National Institutes of Health. Key leadership for the Iowa MDCRC remains the same: Kevin P. Campbell, PhD, is the Director of the Center and Principal Investigator for basic science Project 1; Steven A. Moore, MD, PhD, is Co-Director of the Center and Director of the national shared resource Muscle Biopsy and Cell Culture Repository; and Katherine D. Mathews, MD, is Principal Investigator for the clinical research Project 2. Drs. Campbell, Mathews, and Moore share responsibility for administration and for research training and education.
Muscular dystrophies are a diverse group of inherited disorders characterized by progressive muscle weakness and wasting. The major theme of the University of Iowa MDCRC is to translate research discoveries on the structure and function of dystroglycan into clinical applications for the diagnosis and treatment of patients with dystroglycan-related muscular dystrophy. The overall goal is to explore therapeutic strategies for the treatment of various muscular dystrophies arising from the abnormal processing of dystroglycan (dystroglycanopathies). The specific objectives are to (1) identify mechanistic causes and therapeutic strategies for dystroglycan-related muscular dystrophies and (2) achieve clinical-trial readiness for the dystroglycanopathies. The Center works to achieve these objectives by enabling translational research and providing advanced diagnostic services.
The dystroglycanopathies are a phenotypically and genetically heterogeneous subset of muscular dystrophies caused by hypoglycosylation of alpha-dystroglycan (a-DG). As part of the dystrophin-glycoprotein complex, glycosylated a-DG links the transmembrane b-DG protein to the extracellular matrix, a linkage critical for stability of the muscle membrane, as well as for development of the brain and eye. Currently, 18 genes are known to be required for proper a-Dg glycosylation. Hypoglycosylation of a-DG results in a range of clinical phenotypes, from Walker Warburg syndrome, in which muscular dystrophy is accompanied by severe eye and brain malformations (most severe), through adult-onset limb girdle muscular dystrophy (least severe), and genotype does not reliably predict phenotype.
The Iowa MDCRC is composed of two research projects and three cores. The Center investigators have a proven track record of excellence and collaboration. The Director and Co-director, Kevin Campbell and Steven Moore, are investigators with established records in basic, translational, and clinical research on muscular dystrophy.
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