03 Apr mRNA drug offers hope for treating a devastating childhood disease

By Elie Dolgin, 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 Nature¹, provides a boost to current mRNA applications, which remain limited to vaccines.

“This is a first step in the right direction,” says Katalin Karikó, a Nobel prizewinning pioneer of mRNA technologies who is affiliated with the University of Szeged in Hungary and the University of Pennsylvania in Philadelphia. Read more…