Muscle Cell Biology, Pathophysiology, and Therapeutics

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Abstract This proposal is a competitive renewal application for a multidisciplinary training program in basic and translational research in muscle cell biology at UCLA from a group of highly engaged faculty mentors. The program in Muscle Cell Biology, Pathophysiology, and Therapeutics is focused on bridged training in the fields of muscle and numerous complementary fields and is the sole UCLA training program integrating fundamental laboratory-based research in muscle relevant fields with preclinical and clinical applications. The Muscle Research Center @UCLA is the hub of muscle focused research and educational activities. For many years, the muscle research community at UCLA has been galvanized by funding from two major center grants including the NIH P30 center grant and the NIH U54 Senator Paul D. Wellstone Center of Excellence grant that have been vital to the recruitment of new faculty members from different fields to apply their expertise to skeletal muscle diseases. Our trainees are further developed through activities of a top scoring, NIH-funded Clinical Science Translational Institute (CTSI). A multitude of unique, interactive, and highly integrated activities in muscle research serve as an outstanding resource for our trainees. The training program emphasizes training in the cellular, molecular, and genetic mechanisms of basic muscle function and disease. At both the pre- and postdoctoral levels, the Muscle Cell Biology, Pathophysiology, and Therapeutics main goals are to: 1) identify and support outstanding trainees with a high level of interest and focus in muscle cell biology research, 2) train scientists to conduct cutting-edge research in fundamental and clinical muscle biology, 3) provide trainees with a solid background in the biological sciences with an emphasis in fundamental physiology, immunology, biochemistry, biology, genetics, and now also stem cell biology and genomics, 4) facilitate career development by helping trainees obtain extramural support, and by guiding trainees in obtaining positions in academia and industry and other areas that suit their interests and skills, 5) introduce novel and significant projects for which beneficial outcomes will be derived, 6) acquaint trainees with state-of-the-art research through the training grant- supported fundamental and muscle relevant seminar series and associated journal clubs, and 7) provide trainees with regular opportunities to present their own research in seminar form and receive critical feedback from the training grant faculty and scientific community beyond UCLA at muscle and muscular dystrophy-related national and international meetings, and 8) implement assessments as part of a larger campus effort to develop tools for T32 training programs. UCLA has become a major site for preclinical and clinical trials in muscle disorders, using a variety of innovative approaches. The Neuromuscular Program at UCLA in partnership with the Center for Duchenne Muscular Dystrophy (CDMD) at UCLA has supported over twenty clinical trials in neuromuscular disorders. Thus, trainees are exposed to both basic and clinical aspects of muscle disease research, providing a well-rounded educational experience.