Will microRNAs Ever Succeed in the Clinic

The tiny RNA molecules received Nobel recognition this week, but efforts to turn them into therapeutics have not been easy

This week, the Nobel Committee awarded its Prize in Physiology or Medicine to the two scientists who first characterized microRNAs (miRNAs) 30 years ago. But the small snippets of RNA, which researchers had hoped would fuel a new class of drugs for cancer, heart disease and other conditions, has proven to be a major challenge. So far, no microRNA-based drugs have been approved by the US Food and Drug Administration, with off-target effects and outsized immune responses being two of the reasons why.

However, companies remain interested in harnessing these molecules and the success of drugs that use RNA interference is fueling investment in miRNA drugs against obesity and epilepsy.

To read more about the state of miRNA therapeutics check out this story in Nature.