mRNA drug offers hope for treating a devastating childhood disease
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.
