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 (Dr. Flanigan, Director; Dr. Martin, Co-director; Ashley Madeley, administrator) is responsible for the organization, tracking and reporting of all MDSRC activities.

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Training Core

The Wellstone Training Core (Dr. Harper, Director; Dr. Rafael-Fortney, Associate Director) is centered around the annual Nationwide Children’s/OSU Myology Course, which is provided at no cost to trainees. It currently accepts 70 trainees per year (half clinically based and half lab-based) for an intensive one-week course that includes joint morning lectures followed by afternoon wet lab electives (for PhD trainees) or clinical training (for MDs). To date, more than 770 trainees have attended this course, with many continuing to high profile positions in muscle research. In this Core, we will build upon these successes in order to recruit, train and retain more young investigators in the field of muscular dystrophy research.

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Muscular Dystrophy Cell and Serum Banking Core

The Muscular Dystrophy Cell and Serum Banking Shared Resource Core (Dr. Nicolau, Director; Dr. Flanigan, Associate Director) serves as a resource for each of the projects and for the Wellstone MDSRC network by providing patient-derived fibroblasts, myoblasts and serum samples to both internal and external investigators to serve as tools for basic science discovery and therapeutic development.

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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.