Dr. Diana Bharucha-Goebel is Medical Director of the Gene Therapy Center of Excellence and an investigator in the Center for Gene Therapy within the Abigail Wexner Research Institute at Nationwide Children’s Hospital in Columbus, OH. She is Associate Professor of Neurology at The Ohio State University College of Medicine. She completed her medical training at Drexel University College of Medicine, followed by Pediatric Residency, Child Neurology Residency and Neuromuscular Medicine Fellowship at Children’s Hospital of Philadelphia and Hospital of the University of Pennsylvania. Following her clinical fellowship training, she worked as a clinical research collaborator at the National Institutes of Health (NIH) intramural program and concurrently joined the Division of Neurology at Children’s National Hospital in Washington, DC.  Her research has focused on the development of clinical outcome measures and biomarkers for a rare neurodegenerative disorder, giant axonal neuropathy (GAN). She has worked with Dr. Carsten Bönnemann at NIH on the development and implementation of a first-in-human intrathecal gene transfer trial for GAN. Her primary clinical research interests lie in the development of early phase gene therapy focused trials for patients with rare neuromuscular disorders, including trial readiness through the study of outcome measures and understanding natural history trajectory as it informs trial design. In addition, her research interests have also focused on studying the immunologic consequences of viral mediated gene transfer, and the role of immune modulation in dampening these responses and improving safety of gene replacement approaches. Clinically, Dr. Bharucha-Goebel is a pediatric neuromuscular neurologist providing expert care in the diagnosis, surveillance, management, treatment, and research for children with a wide range of neuromuscular conditions.