Three grants totaling more than $4.5 million, from agencies of the National Institutes of Health, will be used to explore novel treatment strategies for muscular dystrophy.
The grants were awarded by the National Institute of Arthritis and Musculoskeletal and Skin Diseases (NIAMS), National Institute of Neurological Disorders and Stroke (NINDS) and Eunice Kennedy Shriver National Institute of Child Health and Human Development (NICHD) for year one of five-year cooperative agreements.
The grants designate Nationwide Children's Hospital, Columbus, Ohio, as a Senator Paul D. Wellstone Muscular Dystrophy Cooperative Research Center (MDCRC), and continue funding of crucial research by two previously established MDCRCs at the University of Pennsylvania, Philadelphia and the University of Iowa, Iowa City.
Researchers at Nationwide Children's Hospital, under the direction of Jerry Mendell, M.D., will further develop methods to overcome immune barriers to gene correction for Duchenne muscular dystrophy (DMD). A form of muscular dystrophy that affects children and young adults, DMD is caused by an absence of dystrophin, a protein that helps keep muscle cells intact. Gene therapy for DMD includes injecting genes for a functional version of the muscle protein dystrophin, encased in a virus designed to deliver the gene into the muscle cells. In an early trial of this experimental therapy, blood analyses and biopsy slides showed that the immune system in more than half of the children mounted a response to dystrophin or the viral delivery vehicle.
The goal of the research will be to see how many patients have pre-existing immunity to dystrophin that could block the gene transfer. Researchers also will determine if the immune reaction could be circumvented by removing antibodies with a blood-purifying procedure called plasmapheresis or administering drugs that suppress the immune response.
At the University of Pennsylvania, resear
|Contact: Trish Reynolds|
NIH/National Institute of Arthritis and Musculoskeletal and Skin Diseases