Senator Paul D. Wellstone Muscular Dystrophy Cooperative Research Center

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. Currently the program is focused on identifying the underlying processes of inflammation, fibrosis, and fatty infiltration in Duchenne muscular dystrophy, and identifying means to counter them. Project 1, directed by Lee Sweeney at UF, is focused on understanding the pathways and cell types that contribute to inflammation, fibrosis, and fatty deposition, but now will utilize an exciting new mouse model of DMD that may greatly accelerate successful drug translation. Project 2, directed by Elizabeth McNally at Northwestern, is focused on identifying and characterizing modifiers of cardiac disease, which in many cases will be modifiers of skeletal muscle disease as well. In the current application, Project 3, directed by Glenn Walter at UF, will gain insights into how genetic variations in LTBP4 and osteopontin impact imaging biomarkers of disease in the heart, respiratory muscles, and leg muscles of DMD patients. In doing so, Project 3 will develop MRI/MRS protocols for monitoring disease progression in the respiratory muscles. These project are supported by a National Resource Service Core, directed by Elisabeth Barton at UF, that fouses on evaluation of mouse models of DMD. The Wellstone Center also has a Training and Education Core, directed by Andrew Judge at UF.

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