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Iowa State scientists target disease in RNA

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March 25th, 2024 by Ric Hanson

(Iowa News Service) – Researchers at Iowa State University are taking the next step in genetic medicine by targeting diseases in a person’s R-N-A. Most drugs target proteins to interrupt the disease process. Iowa State University researcher Walter Moss says they’ve moved beyond targeting proteins and are taking aim at disease on a smaller level – focusing on ribonucleaic acid, or R-N-A. This will allow scientists to create ‘personalized disease attackers’ that Moss says fit perfectly into ‘pockets’ in a patient’s cells.

“So, there’s the different pockets on a protein drug target. 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 R-N-A 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-percent of the body’s genome is turned into R-N-A as opposed to just 10-percent that becomes protein. He explains 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. 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 R-N-A,” which caused a spike in interest in using R-N-A science more broadly to treat other diseases.