Nationwide Children’s Hospital Receives a Wellstone Muscular Dystrophy Specialized Research Center Award from the NIH

(COLUMBUS, Ohio) - Researchers in the Jerry R. Mendell, MD Center for Gene Therapy at Nationwide Children's Hospital have received a coveted P50 grant from the National Institutes of Health to establish a Wellstone Muscular Dystrophy Specialized Research Center (MDSRC). Named in honor of the late Senator Paul D. Wellstone, a champion of muscular dystrophy research, the Wellstone MDSRC program was established by the National Institutes of Health (NIH) in 2003. One of only six Wellstone awards in the nation, the Nationwide Children's award provides $6 million over four years to establish the center under the leadership of Kevin Flanigan, MD, director of the Center for Gene Therapy at Nationwide Children's, and co-director Paul Martin, PhD.

P50 Center grants from the NIH provide funding to support programs comprising integrated research teams to study a variety of subjects within a central scientific theme. They typically consist of specialized core functions and multiple integrated research projects. The NIH Wellstone program has established a special network of P50 Centers, funded by three different NIH institutes.

The Nationwide Children's Wellstone Center consists of three cores that will share expertise and resources with the Wellstone and broader muscular dystrophy research communities, as well as three projects that will address questions raised or left unanswered by current therapies. All are led by principal investigators within the Center for Gene Therapy at Nationwide Children's.

1. The Administrative Core, led by Dr. Flanigan, will be responsible for outreach and engagement with the patient and research communities.
2. The Cell Line and Tissue Bank Shared Resource Core, led by Nicolas Wein, PhD, provides patient-derived cell lines to researchers at Nationwide Children's and across the Wellstone network, catalyzing the development of new therapies for a wide variety of disease-causing mutations.
3. The Training Core, led by Scott Harper, PhD, provides funding for the annual Nationwide Children's/Ohio State University Myology Course, a unique program which provides an intense introduction to the study of muscle diseases for 60 trainees from across the globe per year.
4. Project 1, led by Dr. Martin, will utilize a novel dual gene AAV vector to both prevent muscle disease and build new muscle function for patients with limb girdle muscular dystrophy type R9 (LGMDR9).
5. Project 2, led by Dr. Flanigan, will develop vectors containing U7 small nuclear RNAs reprogrammed with antisense sequences directed toward splice sites or splice enhancements to restore dystrophin expression.
6. Project 3, led by Nizar Saad, PhD, will study circulating extracellular vesicles to identify novel biomarkers and develop a new generation of gene therapies that increase transduction efficiency, increase dystrophin expression and reduce the development of humoral immunity against adeno-associated virus (AAV) gene therapies.

"This grant funding is testament to the innovative and essential work of our muscular dystrophy research, such as the groundbreaking work that led to the FDA approval of ELEVIDYS for Duchenne muscular dystrophy. P50 Center awards are rare and highly challenging to receive," said Dennis Durbin, MD, MSCE, president of the Abigail Wexner Research Institute at Nationwide Children's. "The Wellstone Muscular Dystrophy Specialized Research Center at Nationwide Children's is one more way we aim to advance the care of children with muscular dystrophy here in central Ohio and across the U.S."

"We are grateful to the National Institute of Child Health and Human Development for providing us with this support," adds Dr. Flanigan. "Our research and clinical teams look forward to expanding our engagement with the research and patient communities to the benefit of all."

About The Abigail Wexner Research Institute at Nationwide Children's Hospital
Named to the Top 10 Honor Roll on U.S. News & World Report's 2024-25 list of "Best Children's Hospitals," Nationwide Children's Hospital is one of America's largest not-for-profit free-standing pediatric health care systems providing unique expertise in pediatric population health, behavioral health, genomics and health equity as the next frontiers in pediatric medicine, leading to best outcomes for the health of the whole child. Integrated clinical and research programs are part of what allows Nationwide Children's to advance its unique model of care. As home to the Department of Pediatrics of The Ohio State University College of Medicine, Nationwide Children's faculty train the next generation of pediatricians, scientists and pediatric specialists. The Abigail Wexner Research Institute at Nationwide Children's Hospital is one of the Top 10 National Institutes of Health-funded free-standing pediatric research facilities in the U.S., supporting basic, clinical, translational, behavioral and population health research. The AWRI is comprised of multidisciplinary Centers of Emphasis paired with advanced infrastructure supporting capabilities such as technology commercialization for discoveries; gene- and cell-based therapies; and genome sequencing and analysis. More information is available at NationwideChildrens.org/Research.

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