Center for Gene Therapy :: The Research Institute at Nationwide Children's Hospital

Gene Therapy

The mission of the Center for Gene Therapy is to investigate and employ the use of gene and cell based therapeutics for prevention and treatment of human diseases including: neuromuscular and neurodegenerative diseases, lysosomal storage disorders, ischemia and re-perfusion injury, neonatal hypertension, cancer and infectious diseases.

Learn about our areas of focus and featured research projects.

Featured Video: Treating Spinal Muscular Atrophy Type 1 with Gene Therapy - Tenley's Story

Wellstone Muscular Dystrophy Cooperative Research Center

The National Institutes of Health has designated the Center for Gene Therapy as a Paul D. Wellstone Muscular Dystrophy Cooperative Research Center (MDCRC). MDCRCs promote basic, translational and clinical research and provide important resources that can be shared within the national muscle biology and neuromuscular research communities.

The MDCRC will allow Nationwide Children's researchers to further develop methods to overcome immune barriers to gene correction for Duchenne muscular dystrophy.

View the Nationwide Children's Wellstone Center page.

Viral Vector Core & Clinical Manufacturing Facility

The Center for Gene Therapy and the Viral Vector Core are home to a Good Manufacturing Practice (GMP) production facility for manufacture of clinical-grade rAAV vectors. 

View the Viral Vector Core & Clinical Manufacturing Facility site.

The Ohio State University and Nationwide Children's Muscle Group

The OSU and Nationwide Children's Muscle Group brings together investigators with diverse research interests in skeletal muscle, cardiac muscle, and neuromuscular biology.

Muscular Dystrophy Podcast

Hosted by Kevin Flanigan, MD, "This Month in Muscular Dystrophy" podcasts highlight the latest in muscular dystrophy and other inherited neuromuscular disease research. During each podcast, authors of recent publications discuss how their work improves our understanding of inherited neuromuscular diseases, and what their work might mean for treatment of these diseases.

Neuromuscular Disorders Program at Nationwide Children's

Nationwide Children's Hospital
700 Children's Drive Columbus, Ohio 43205 614.722.2000