Wellstone Center Senator Paul D. Wellstone Center at the UF Center for Neurogenetics
The mission of our Wellstone Center, a long-term collaboration between the University of Florida and the University of Rochester, is to perform cutting-edge research that leads to transformative therapies for the most prevalent form of adult-onset muscular dystrophy, myotonic dystrophy.
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For Patients
The Registry’s main goals are to advance research and knowledge of myotonic dystrophy (DM) and facioscapulohumeral muscular dystrophy (FSHD).
About the center
Our Center is based on a long history of creating and maintaining exceptionally close research interactions between basic scientists at the University of Florida and clinical researchers at the University of Rochester. The three scientific projects are highly synergistic and center on two themes: 1) continuing to accelerate and support clinical trials in the DM1 space, including preparing for a potentially imminent post-approval landscape; 2) accelerating both basic science and translational efforts for DM2 so that DM2 can also rapidly advance to clinical trials. Project 1 (Drs. Ranum and Swanson) focuses on DM2 mouse models that we have recently generated, including the first human BAC transgenic mouse multisystemic model for DM2. Project 2 (Dr. Wang) objectives are to clarify molecular mechanisms underlying several leading therapeutic approaches using new Dmpk CTG expansion mouse knockin multisystemic models for DM1. Project 3 (Drs. Hamel and Thornton) transitions to current clinical issues by analyzing a large DM1 cohort using a remote assessment strategy to examine disease severity. A DNA Bank will also be established for comprehensive analysis of expanded repeats and statistical models will be developed to study relationships between repeat length, age, sex and disease severity. For DM2, remote patient assessments and targeted recruitment will facilitate studies designed to assess roles for RAN translation and RNA toxicity. In combination with these projects, our Administrative Core (Drs. Swanson and Thornton) will provide rigorous oversight for all projects and cores and emphasize synergy between the projects and cores. We will continue to expand our Shared Resource Core (Dr. Hamel) since this core is an essential resource for our Wellstone Projects but also serves the broader DM1 and DM2 research and clinical community focused on dominant muscular dystrophies. Our Training Core (Dr. Wang) is designed to attract and build competency in the next generation of basic and clinical investigators focused on DM1 and DM2 by training in wet and dry lab settings and will provide training in communication skills needed to effectively engage all DM stakeholders.