Visualizing Gene Therapy for SMA

Visualizing Gene Therapy for SMA 150 150 Abbie Miller

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.

About the author

Abbie (Roth) Miller, MWC, is a passionate communicator of science. As the manager, medical and science content, at Nationwide Children’s Hospital, she shares stories about innovative research and discovery with audiences ranging from parents to preeminent researchers and leaders. Before coming to Nationwide Children’s, Abbie used her communication skills to engage audiences with a wide variety of science topics. She is a Medical Writer Certified®, credentialed by the American Medical Writers Association.