This MDCRC application is designed to advance our abilities to treat the muscular dystrophies by capitalizing on the breadth and depth of expertise at our collaborating institutions in the fields of neuromuscular disease research and patient care, molecular genetics, immunology, and gene therapy. The participating researchers include scientists and clinicians from the University of Washington Medical Center, Children's Hospital and Regional Medical Center, Fred Hutchinson Cancer Research Center, and the VA Medical Center, Seattle. Our goals are to provide an interactive environment that will advance knowledge of and treatments for the muscular dystrophies by promoting collaborations, providing shared resources, advancing basic research, stimulating translational research, fostering outreach activities, developing greater patient awareness of basic and clinical research and enabling their participation in clinical trials, and by facilitating the development gene therapy for the muscular dystrophies. Our Center is composed of four research projects and three Core laboratories, and includes a membership of more than 30 faculty members in the Seattle area with interests in neuromuscular disease research. The director and codirector are J Chamberlain and S Tapscott, researchers with a long history of genetic and clinical studies of the muscular dystrophies. Project 1 (J Chamberlain, S Tapscott and S Froehner) will conduct pre-clinical studies in dystrophic mice that lead to a phase I clinical tdal of gene therapy for Duchenne muscular dystrophy (DMD). Project2 (M-T Little and S Tapscott) will study the canine model for DMD to address pre-clinical issues related to DMD therapy by emphasizing immunological, safety and allometdc issues important for the evelopment of therapies for DMD. Project 3 (S Hauschka) explores issues important for regulating gene expression vectors in muscles of humans, mice and dogs, and will develop animal and cell culture models for human muscle gene transfer. Project 4 (S. Tapscott) will study abnormal gene expression in cells from individuals with myotonic dystrophy, and will explore the clinical significance of methylation at the DM1 locus in adult onset myotonic dystrophy. Core A (Chamberlain) is an administrative core to coordinate and organize collaborative and interactive activities within and outside of our center. Core B (Chamberlain, J Allen) is a viral vector core and a scientific research resource core that will facilitate the availability of research and clinical grade gene transfer vectors for basic research and the development of gene therapies for the muscular dystrophies. Finally, Core C (T Bird) is a genetic counseling core for patients and families with muscular dystrophy that will also allow increased participation of patients in studies of a variety of types of muscular dystrophy.
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