One Gene, Big Impact: The Science Behind a New Therapy for Brain and Muscle Health
One Gene, Big Impact: The Science Behind a New Therapy for Brain and Muscle Health https://pediatricsnationwide.org/wp-content/uploads/2026/02/Original_hi_res_JPG-091725SC0193_Color-1024x683.jpg 1024 683 Madison Storm https://pediatricsnationwide.org/wp-content/uploads/2023/09/092023BT159.png
A promising new gene therapy developed at Nationwide Children’s Hospital is offering hope for new patients with neurodegenerative and muscle disorders. Led by Zarife Sahenk, MD, PhD, a neurologist, principal investigator in the Jerry R. Mendell Center for Gene Therapy and director of Clinical and Experimental Neuromuscular Pathology at the Abigail Wexner Research Institute, the…