The purpose of this Administrative Supplement is to maintain the three cores of the UF Wellston Center during its one-year no cost extension in order to serve the administrative, training, and resource functions of the Center, as well as the national muscular dystrophy community. The Administrative Core provides integration and management of activities for the Center. It promotes interactions and communications between the research and patient/advocacy communities on both a national and local level. The Administrative Core maintains our website to communicate the Center mission and the availability of training opportunities and services available through our Shared Scientific Resource Core. The overall objective of the Training Core is to increase the number of rigorously trained and scientifically competent researchers capable of sustaining productive basic science, translational and/or clinical research programs in muscular dystrophy. To meet this central objective, the core will identify and recruit qualified basic science and clinical predoctoral and postdoctoral candidates who demonstrate a genuine interest in, and potential for, independent basic science, translational and/or clinical research in muscular dystrophy. Particular emphasis will be placed on the recruitment of trainees from underrepresented groups with respect to race, ethnicity, gender, socio-economic status and disabilities. Critical to the evaluation of potential therapeutics (pharmacological, gene, or cell therapies) are sensitive and repeatable physiological assessments of muscle function applied to mouse models of dystrophy. Therefore, we are proposing to continue our Shared Scientific Resource Core that performs ex vivo, in situ, and whole animal assessments of muscle integrity and function. This core will not only support the needs of the projects within the center during the no cost extension, but will also serve as a national resource for performing functional evaluation of potential therapies for the muscular dystrophies. The Physiological Assessment Core / Shared Scientific Resource Core is housed in the laboratory space designated to Dr. Barton, the Core Director. Dr. Barton is a muscle physiologist with extensive experience evaluating muscle function in mice. This includes all instrumentation for muscle physiological measurements, specialized equipment for hindlimb suspension experiments, documented free wheel running, and treadmill running, as well as interim storage of samples in both ?80C and liquid nitrogen freezers. We propose to collaborate with neighboring colleagues to assess whole animal respiratory function, which will complement ex vivo assessment of the diaphragm. We believe that this Core has provided a previously unmet need in the past 15 years of its existence, and will continue to be a valuable resource that will enable the entire muscular dystrophy research community to utilize these assays to assess the potential benefits of a large number of approaches to the treatment of different forms of muscular dystrophy.
The goal of this Wellstone Muscular Dystrophy Specialized Research Center is to identify emerging therapeutic targets and therapeutics, as well as to develop imaging tools to monitor the efficacy of therapies, that control inflammation, fibrosis, and disease progression in a number of muscular dystrophies. We also are focused on understanding the mechanisms and unwanted side effects of current therapies (e.g. corticosteroids) in order to improve upon their efficacy and use. With the dawn of gene therapy for Duchenne muscular dystrophy, we feel it is imperative to understand the deficiencies of the emerging treatments in order to improve upon them before the deficiencies become evident in patients.
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