Skip to Main Content

Gene therapies hold huge promise for inherited diseases that attack the nervous system — but studies show they can inadvertently damage sensitive nerve cells, triggering side effects like weakness, numbness, and intense pain.

University of Pennsylvania scientist and gene therapy pioneer James Wilson has developed a new tactic designed to make these treatments safer for the nervous system. The approach, detailed in a paper published Wednesday in Science Translational Medicine, involves modifying the viral vectors used in gene therapy so they don’t harm delicate nerve clusters as they work to correct disease.

Unlock this article by subscribing to STAT+ and enjoy your first 30 days free!