Federal Grant Award Summary

The National Institute of Neurological Disorders and Stroke (NINDS) awarded $341,129 to the Research Institute at Nationwide Children's Hospital for a Training Program in Basic and Translational Gene Therapy Research, effective July 1, 2026, through June 30, 2031, under the Extramural Research Programs in the Neurosciences and Neurological Disorders program (CFDA 93.853). The training program will prepare the next generation of clinicians and scientists in gene therapy development and clinical translation, leveraging the Center for Gene Therapy's established expertise in advancing FDA-approved therapies for spinal muscular atrophy and Duchenne muscular dystrophy. The program addresses a critical workforce gap by providing comprehensive instruction in emerging gene therapy technologies, including base editing, prime editing, RNA interference, exon skipping, and advanced viral and non-viral delivery approaches, with oversight from a cohort of 20 mentors and co-mentors who are leaders in the gene therapy field.

The training program will equip participants with specialized knowledge in managing adverse events associated with adeno-associated virus (AAV) gene therapies, navigating the unique scientific and ethical challenges inherent in human gene therapy development, and implementing clinically proven strategies alongside innovative emerging technologies. By addressing the growing clinical availability of gene therapies and the corresponding demand for well-trained personnel across multiple disease types and therapeutic modalities, the program supports NINDS's mission to reduce the burden of neurological disease through advanced research training and workforce development in this critical therapeutic area.