mRNA Drug Offers Hope for Treating a Devastating Childhood Disease

April 3, 2024

CGI of an RNA molecule

(Nature) – A drug that uses messenger RNA technology has shown early success in addressing the core deficiency behind a rare genetic disorder. The results have ignited hope that the technology — which first gained attention through its breakthrough use in COVID-19 vaccines — could realize its long-awaited promise of generating therapeutic proteins directly in the body.

This clinical advance, reported today in Nature, provides a boost to current mRNA applications, which remain limited to vaccines. (Read More)