Skip to Main Content

On Friday, the Food and Drug Administration approved the first-ever drug to rely on a Nobel-prize-winning technique that mutes disease-causing genes — a watershed moment for that field of research, and a starting pistol for the race to find a way to use the therapy for other debilitating genetic diseases like ALS or Huntington’s.

But already, a major question looms: What if the technique won’t work in other parts of the body?

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