Wellstone Muscular Dystrophy Specialized Research Center (MDSRC)

The National Institutes of Health (NIH)-funded Wellstone Muscular Dystrophy Specialized Research Center (MDSRC) network, named for the late Senator Paul D. Wellstone of Minnesota, is a group of collaborative, NIH-funded, centers throughout the United States committed to basic, translational and clinical research on various forms of muscular dystrophy. Wellstone Centers seek to promote collaborative basic, translational and clinical research and provide important resources that can be used by muscular dystrophy researchers.

Wellstone leadership team standing on a staircase.

Who We Are

The Wellstone Center at the Abigail Wexner Research Institute at Nationwide Children’s Hospital, directed by Kevin Flanigan, MD, and co-directed by Paul Martin, PhD, is proud to be a part of the Wellstone network. The goal of our MDSRC is to develop the next directions in gene therapy for the treatment of children with muscular dystrophy, including children with Duchenne Muscular Dystrophy (DMD), Limb Girdle Muscular Dystrophy (LGMD) and Facioscapulohumeral Muscular Dystrophy (FSHD).

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Muscular Dystrophy Specialized Research Center Cores

Administrative Core

The Administrative Core, under the direction of Kevin Flanigan, MD and Paul Martin, PhD monitor and oversee core resource utilization and facilitating the center’s outreach program to the muscular dystrophy community.

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Training Core

The MDSRC’s Training Core, led by Scott Harper, PhD, and Jill Rafael-Fortney, PhD, facilitates the training of new scientists in muscular dystrophy research, including sponsoring the highly regarded annual Nationwide Children’s/Ohio State University Myology Course for medical and scientific postdoctoral fellows.

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Muscular Dystrophy Cell and Serum Banking Core

Led by Stefan Nicolau, MD, and Kevin Flanigan, MD, the core characterizes and provides cells and serum from muscular dystrophy patients with defined genetic mutations to investigators worldwide.

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MDSRC Affiliations

The Nationwide Children’s Wellstone Center is affiliated with the nationally-recognized Nationwide Children’s Neuromuscular and Muscular Dystrophy Clinics as well as with the Jerry Mendell Center for Gene Therapy and The Ohio State University. The center is committed to meaningful engagement with patients and families affected by muscular dystrophies.

Research Projects

Project 1: Dr. Martin will pioneer new dual gene therapy approaches where gene replacement therapy, to suppress subsequent disease, is coupled with a muscle-building therapy to help patients regain lost muscle mass and strength. Click here to learn more.
Project 2: This project, led by Dr. Flanigan, will develop new AAV-based approaches to stimulate exon skipping of dystrophin gene transcripts to treat patients with DMD. Click here to learn more.
Project 3: This project, led by Nizar Saad, PhD, will focus on defining new biomarkers for DMD and FSHD in muscle extracellular vesicles (EVs). It will also develop methods to encapsulate AAV treatments within EVs to increase immune evasion and treatment efficacy. Click here to learn more.