Visualizing Gene Therapy for SMA

Spinal muscular atrophy (SMA), a degenerative neuromuscular disease, is the most common genetic cause of death for infants. Virtually all children affected with SMA type 1 die by 2 years of age. In 2014, Jerry Mendell, MD, director of the Center for Gene Therapy in the Research Institute at Nationwide Children’s Hospital, began a phase 1 gene transfer clinical trial for SMA type 1. In this trial, a large number of modified viruses containing the missing SMN1 gene are administered to the patient. By administering the gene early in the disease process, the team hopes to effectively halt the progression of the disease. Nationwide Children’s has done more gene therapy clinical trials for neuromuscular disease than any place in the world. The current trial is providing hope to a patient named Tenley and other families affected by SMA. Watch the video below for her story and a brief animation of the gene therapy procedure.