Summary
In December 2017 the U.S. Food and Drug Administration (FDA) announced approval of a novel gene therapy, Luxturna, to treat patients with a rare form of inherited vision loss. It is the first gene therapy approved in the United States to target a disease caused by mutations in a specific gene. In this case, the RPE65 gene, which affects vision. For Dr. Jean Bennett, the physician scientist behind this medical breakthrough, being able to change the prognosis for people who are blind or losing their vision — and to see the profound impact that this has on their life — has been a career well spent.
