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.
Meet Our Team
Learn more about director Kevin Flanigan, MD, and the team of principal investigators at the Center for Gene Therapy.
Gene Therapy Research and Projects
Learn more about muscular dystrophy research and other areas of focus at the Center for Gene Therapy and see the latest featured research and projects to come out of our center.
The FDA issued an approval for the gene therapy for SMA developed at Nationwide Children's, laying the groundwork for it to change the lives of children all over the country.
Gene Therapy and Neuromuscular Training
Nationwide Children’s Hospital is not only home to world-class neuromuscular research and clinical services, it’s also the hub of an extraordinary and collaborative training environment.
Annual Myology Training Course
The Myology Course at Nationwide Children's is the only U.S. workshop of its kind, featuring a mix of basic and clinical science lectures followed by specific training in either clinical treatment protocols or basic science laboratory techniques.
OSU 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.
Gene Therapy Clinical Research
Learn more about ongoing clinical trials and other clinical research related to muscular dystrophies and other disorders.
The Bradbury Laboratory is dedicated to understanding disease mechanisms and developing therapeutic approaches for rare, pediatric neurodegenerative disorders.
As part of the Center for Gene Therapy the Lowes Lab supports clinical trials in rare diseases.
The Rashnonejad Lab focuses on developing adeno-associated virus (AAV)-based gene therapies for neurodegenerative and neuromuscular diseases.
Nicolas Wein, PhD (Assistant Professor) and his group study the mechanisms of pathogenesis of neuromuscular disorders with a primary focus on Duchenne Muscular dystrophy (DMD) and Dystrophy myotonic type 1 (DM1).
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.