News Health & Medicine

The first gene-silencing drug wins FDA approval

Using RNA interference, patisiran prevents symptoms by blocking DNA instructions

illustration of RNA

INTERCEPTION  Small bits of RNA can latch onto longer messenger RNA molecules that are carrying DNA’s instructions to make proteins (illustrated), halting the production process. A newly approved drug uses this natural process to turn off a specific gene.

BSIP SA/Alamy 

By Laurel Hamers

- More than 2 years ago

A Nobel Prize–winning discovery — that small double-stranded RNA molecules can silence genes by interrupting the translation of DNA’s instructions into proteins — is finally delivering on its medical promise.