Gene Therapy :: Nationwide Children's Hospital

Gene Therapy Clinical Studies

Investigators at the Center for Gene Therapy in The Research Institute at Nationwide Children's Hospital are currently conducting numerous clinical research studies, described in detail below. The Neurosciences Center at Nationwide Children's offers patients and families a comprehensive approach to the care for children and adolescents with disorders of the brain, spine and nervous system; from initial diagnosis and treatment to rehabilitation and long-term follow-up care.

Learn more about the SMA type 1 clinical trial. Please note: this trial is not recruiting participants at this time. Information for parents of children with SMA1 who want to learn more about study participation can visit:

Batten Disease

Congenital Myasthenic Syndromes (CMS)

Duchenne Muscular Dystrophy (DMD)

Limb Girdle Muscular Dystrophy (LGMD)

Mucopolysaccharidosis (MPS) IIIA


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