Center for 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.

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The Research Institute is ranked among the top 10 for NIH funding among free-standing children's hospitals.

GMP Facility

The Research Institute at Nationwide Children’s Hospital is the home to a current Good Manufacturing Practices (cGMP) Clinical Manufacturing Faculty that operates according to FDA cGMP Guidelines to ensure the safety of manufactured biologic products.

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