UCLA Muscular Dystrophy Core Center

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DESCRIPTION (provided by applicant): The Center for Duchenne Muscular Dystrophy ("CDMD") provides an academic home in Los Angeles for the conduct of translational research for the muscular dystrophies. Although CDMD supports all muscle and muscular dystrophy related research, its primary focus is Duchenne Muscular Dystrophy (DMD), the most common lethal genetic disorder in children. CDMD unites recognized leaders in basic and clinical muscular dystrophy research with the immense technological and educational infrastructure of UCLA to facilitate discovery and development of new therapeutics for degenerative muscle disease. Our location in Los Angeles, the most populous county in the United States, gives us access to a huge catchment from which to recruit patients with rare muscle diseases studied at CDMD. These resources, coupled with the successes of our existing Center, position CDMD to become the leading translational muscular dystrophy center on the West Coast, if not the nation. Since the first funding of the CDMD Core Center in 2009, the Center has successfully expanded the number of multidisciplinary collaborations focused on muscular dystrophy research and accelerated the discovery and testing of potential therapeutics. In the past four years, we have awarded $650,000 in pilot and feasibility seed grants to 17 labs, and 12 of which went to investigators new to muscle or muscular dystrophy research. Membership in CDMD has doubled to nearly 45 members. Researchers affiliated with our Center have initiated 13 clinical trials and preclinical tests of four therapeutic targets. In this application, we propose to furthe expand our infrastructure and services to create an ecosystem that supports translation of discoveries in muscle and muscular dystrophy research. Through an Administrative Core, an Enrichment Core and 3 basic science cores, CDMD will provide the institutional resources necessary for all aspects of basic science discovery, pre-clinical testing, and clinical trials. Th basic science cores, which include a High Throughput Screening and Cell Repository Core (Core B), a Muscle Phenotyping and Imaging Core (Core C), and a Bioinformatics and Genomics Core (Core D) are designed to facilitate discovery of new therapeutic targets, novel compounds and create models to test these compounds and targets related to muscle disease.