Diana Bharucha-Goebel, MD

Nationwide Children’s Hospital and
The Ohio State University College of Medicine
Columbus, OH

Dr. Diana Bharucha-Goebel is Medical Director of the Gene Therapy Center of Excellence (COE) and Associate Professor of Pediatrics at Nationwide Children’s Hospital and at The Ohio State University College of Medicine. She works on the evaluation of disease-focused outcome measures and trial design for neuromuscular diseases and studies the impact of novel therapies on care needs and emerging phenotypes for pediatric neuromuscular disorders. Following work co-leading a first-in-human intrathecal AAV-mediated gene transfer trial for giant axonal neuropathy (GAN), she also focuses on investigating how vector genome dose, capsid, route, and immune modulation regimens may impact the safety, clinical, laboratory, and immunologic profile in patients following viral mediated gene transfer targeting the peripheral nervous system and musculoskeletal diseases.