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Senator Paul D. Wellstone Muscular Dystrophy Cooperative Research Center

The Research Institute at Nationwide Children’s Hospital launched a full scale attack on Duchenne and Becker Muscular Dystrophy.  This is part of the effort established through the MD-Care Act.

The MD-Care Act (established through Public Law 107-84) specified provisions for expanding and intensifying research on muscular dystrophy.  The Law further mandated that NIH establish centers of excellence for research in muscular dystrophy.  These Centers were developed in honor of Senator Paul D. Wellstone, a champion of muscular dystrophy research and designated “The Muscular Dystrophy Cooperative Research Centers (MDCRCs).

The NIH sponsors of the MDCRCs include The National Institute of Neurological Disorders and Stroke (NINDS), the National Institute of Arthritis and Musculoskeletal and Skin Diseases (NIAMS), the National Institute of Child Health and Human Development (NICHD), and the National Heart, Lung, and Blood Institute.  The MDCRCs are composed of a multidisciplinary mixture of basic, translational, and clinical studies that are tightly integrated to foster the development of new therapies for the muscular dystrophies.

No matter how successful we are in treating the skeletal muscle complications of muscular dystrophy, the final outcome for patients will be determined by successful treatment of the disease.  The collaborative program at the Research Institute at Nationwide Children’s Hospital is committed to finding a treatment in muscular dystrophy.

The key elements of the program include:

1. Identify the prevalence and molecular determinants of immunity to dystrophin epitopes
2. Determine what role immune responses to both dystrophin and recombinant AAV may play in the success or failure of gene correction therapies
3. Characterize the properties and prevalence of dystrophin-specific T-cells in DMD subjects
4. Determine the effect of initiation of glucocorticoid therapy on modulating immune response
5. Assess immune responses in a pilot trial of vascular delivery of an AAV8.MCK.micro-dystrophin.
6. Assess enhancement of transgene delivery in AAV-immune individuals
7. Determine if inhibition of T cell activation and expansion facilitates persistent expression of a non-self transgene