The Shared Resource Core of the Wellstone/Myology Institute was established under the Senator Paul D. Wellstone Muscular Dystrophy Cooperative Research Center, which was awarded to Dr. H. Lee Sweeney (overall PI) and the University of Florida in 2015. The Physiological Assessment Core is under the direction of Dr. Elisabeth Barton, and is intended to serve as a national resource for performing functional evaluation of potential therapies for the muscular dystrophies. The mission of the Core is to evaluate therapeutics interventions in mouse models in disease to aide academics and industry in making decisions as to which of their therapeutics should move forward to clinical trials.
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. The Core performs ex vivo, in situ, and whole animal assessments of muscle integrity and function, and maintains in-house colonies of mouse models of DMD and LGMD.
The long-term goal of the UF Wellstone Muscular Dystrophy Cooperative Center (MDCRC) is to promote the translation of promising emerging therapeutic targets into clinical trials for muscular dystrophy patients. As such, there are discovery efforts for new therapeutic targets, ongoing examinations of possible therapeutics and their impact on disease models of muscular dystrophy, and efforts to build tools necessary for human clinical trials in the muscular dystrophies. The Core expands upon these goals by offering the physiological assessments and colony services to fellow academics as well as industry. Promoting translational research takes basic discoveries into clinical trials to advance the understanding of and combat human diseases.