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.
The first drug that takes advantage of this natural biological process, called RNA interference, was approved August 10 by the U.S. Food and Drug Administration. It targets a rare hereditary disease that causes misshapen proteins to build up in patients’ nerves, tissues and organs, causing loss of sensation, organ failure and even death.
Heredity transthyretin amyloidosis, or ATTR, affects about 50,000 people worldwide. This drug will help the subset of those patients who have neurological impairments.
Called patisiran, the drug uses specially designed pieces of RNA to silence a mutated gene that, when active in the liver, is responsible for patients’ symptoms. In a recent 18-month clinical trial, patients who received patisiran injections every three weeks showed a slight decrease in neurological symptoms, whereas patients on the placebo worsened overall. It’s not a cure — people still have the genetic mutation — but the treatment prevents the disease from progressing.
This approval is “just the beginning,” says Craig Mello of the University of Massachusetts Medical School in Worcester, who co-discovered the process of…
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