Groundbreaking Gene Therapy Offers Hope for Rare Disease and Neurological Disorders

Steven Gray, PhD

A gene therapy developed by UT Southwestern Medical Center researchers for the rare disease giant axonal neuropathy (GAN) was found to be well tolerated in pediatric patients and showed significant benefits, according to a new study published in the New England Journal of Medicine. This phase one clinical trial marks the first of its kind for any disease, employing an approach to deliver a therapeutic gene broadly to the brain and spinal cord via intrathecal injection. Co-author Steven Gray, Ph.D., Associate Professor of Pediatrics, Molecular Biology, Neurology, and in the Eugene McDermott Center for Human Growth and Development at UT Southwestern, highlighted the clear and statistically significant benefits observed in patients, offering hope not only for those with GAN but also for individuals with various other neurological disorders.