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 … Continue reading →