Iowa State scientists target disease in RNA

AMES — Researchers at Iowa State University are taking the next step in genetic medicine by targeting diseases in a person’s RNA. Most drugs target proteins to interrupt the disease process.

Iowa State University Assistant Professor of Biochemistry Walter Moss says they’ve moved beyond targeting proteins, and are taking aim at disease on a smaller level – focusing on ribonucleic acid, or RNA. This will allow scientists to create ‘personalized disease attackers’ that Moss said fit perfectly into ‘pockets’ in a patient’s cells. “So, there’s the different pockets on a protein drug target,” said Moss. “You’d be able to get that drug into that pocket, either to interfere with the protein function or the protein interaction in some way. And the same idea would be with RNA.”

Moss says the RNA approach could be used to target cancer and genetic diseases, but adds years of testing remain before the technology will be ready for the commercial market. Moss says 90% of the body’s genome is turned into RNA as opposed to just 10% that becomes protein. He explained that dramatic ratio presents scientists with new opportunities.  “It opens up whole new avenues of targeting diseases that aren’t currently possible at the protein level,” said Moss. “Because now you can start to treat RNA-related diseases, or diseases where there is no good protein drug target.”

Moss says COVID vaccines harnessed the power of what’s known as “messenger RNA,” which caused a spike in interest in using RNA science more broadly to treat other diseases.