This proposal is a new 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. For many years, the UCLA Center for Duchenne Muscular Dystrophy (CDMD) has been vital to the recruitment of new faculty members from different fields to apply their expertise to skeletal muscle diseases. The CDMD has leveraged UCLA's internationally recognized standing to become a major center in DMD preclinical and clinical trials that use a variety of innovative approaches including exon skipping and PDE5 inhibitors. We have garnered significant matching funds from intramural and extramural sources to further advance our training mission. In addition, our trainees are further developed through activities and development of a top scoring, NIH-funded Clinical Science Translational Institute (CTSI). Thus, the training program provides as a strong complement of basic, translational, and clinical investigations in molecular mechanisms of muscular dystrophy at UCLA. 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 muscular dystrophy, with a focus on Duchenne muscular dystrophy as a model. 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 of the training program as well as trainees for dissemination to the science and education community at large.
The field of muscular dystrophy translational research is expanding as academic and industry partnerships are yielding new potential treatments that are undergoing assessment in the clinics. Well-trained and highly motivated investigators in this field are essential for the progress of basic and translational muscle cell research as there have been major breakthroughs in mechanisms of disease and the emergence of new potential therapies for muscular dystrophy. Trainees of the Muscle Cell Biology, Pathophysiology, and Therapeutics Program will fulfill its stated mission by advancing to positions of leadership in academia or industry and continuing to uncover basic principles in mechanisms of disease while also developing next- generation therapies based in muscle diseases for the betterment of patients and their families.
