The nominating institution Harvard Medical School on the project:
If there is a worse disease than ALS, you should hope never to see it. Relentlessly progressive and 100% fatal, it has no treatment. By means of a clever genetic screen, the nominee and his colleagues identified a gene that, when modestly overexpressed, is able to rescue nerve cell models of ALS. In the most severe animal models, it can also halt disease progression. This novel gene therapy has identified a new pathway for treating ALS, and is scheduled to begin human clinical trials sometime in 2021.
Tags: Bio-Tech, Med-Tech, Resilience, Trust in science, Human Health