Center for Gene Therapy :: The Research Institute at …

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.

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.

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.

Investigators with the Center for Gene Therapy currently are conducting numerous clinical research studies, especially for neuromuscular disorders.

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

Learn how the 24 labs within OSU/Nationwide Children's Muscle Group are working to improve approaches to treat muscle injury and disease. Read about how their collaborations are changing the way we treat neuromuscular diseases.

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.

Parent Project Muscular Dystrophy, an advocacy group founded by parents and family members of patients with Duchenne muscular dystrophy, recently submitted the first-ever patient-initiated guidance to the U.S. Food and Drug Administration for pharmaceutical companies to help expedite drug development for Duchenne.Kevin Flanigan, MD, principal investigator in the Center for GeneTherapy at The Research Institute, specializes in inherited muscular disorders and their potential therapies, and chaired one of seven working groups on the steering committee that drafted the guidance.

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Center for Gene Therapy :: The Research Institute at ...