The award explores and refines therapeutic strategies that have shown promise in animal models of muscular dystrophy. Studies include further research in the laboratory as well as clinical trials to assess efficacy in patients. These efforts include replacing defective genes responsible for muscular dystrophy with healthy ones. Other trials use substitute or surrogate genes to restore muscle function. A third approach attempts to repair gene function using specially designed medications. All the studies aim to slow the progression of muscular dystrophy.
In 2001, the United States MD-CARE Act specified a number of provisions for expanding and intensifying research on muscular dystrophy, notably the NIH’s establishment of centers of excellence for research on these muscle diseases. The Muscular Dystrophy Cooperative Research Centers (MDCRCs) program was subsequently developed in honor of Senator Paul D. Wellstone, a champion of muscular dystrophy research. MDCRCs promote basic, translational and clinical research and provide important resources that can be shared within the national muscle biology and neuromuscular research communities. The MDCRCs also serve as focal points for research collaborations and provide training and guidance for basic and clinical researchers.
The MDCRC will allow Nationwide Children's researchers to further develop methods to overcome immune barriers to gene correction for Duchenne muscular dystrophy (DMD). Jerry Mendell, MD, director, Center for Gene Therapy and Chris Walker, PhD, director, Center for Vaccine and Immunity, are co-leaders for the new center.
“Nationwide Children's has been built an impressive history of innovative research and scientific collaboration with the goal of curing muscular dystrophies,” said Dr. Mendell. Within the past five years, researchers in the Center for Gene Therapy and Center for Vaccine and Immunity have been awarded multi-million-dollar grants from the NIH, and published key articles in high-impact journals such as Cell, the Proceedings of the National Academy of Sciences and Nature. The Muscular Dystrophy Association (MDA) named Nationwide Children's Hospital to its Clinical Research Network in 2008. “The designation as a Wellstone Center reiterates that we’re moving in the right direction,” said Dr. Mendell.
“Receiving this grant and designation as a Wellstone Center – a prestigious national validation of the groundbreaking muscular dystrophy research being performed by our neurosciences group – establishes Nationwide Children’s as a national leader in neuromuscular research and treatment,” said John Barnard, MD, president of The Research Institute at Nationwide Children’s Hospital. “I am especially pleased that a major strength of our research program is it multidisciplinary nature, including intensive collaborations with colleagues in the Center for Vaccines and Immunity here at The Research Institute and in various departments on The Ohio State University’s main campus.”