mRNA drug offers hope for treating a devastating childhood disease

Elie Dolgin • April 3, 2024

Drug trial results show that vaccines aren't the only use for the mRNA technology behind the most widely used COVID-19 jabs.


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.

“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.

Yet challenges remain — especially the fleeting nature of mRNA and the side effects it causes, which complicate the path towards widespread adoption.


Continue reading at Nature.

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