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Can we cure diseases by drugging our RNA?

Science on Tap question, answered
October 30, 2020
Science question

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October's Science on Tap speaker was structural biologist Karissa Sanbonmatsu of Los Alamos National Laboratory. In this livestreamed conversation with Museum Director Linda Deck, she shared her team's latest discoveries about potential treatments for heart conditions using long, non-coding RNA.

Q: Can we cure diseases by drugging our RNA?

A: The short answer is yes, scientists believe we can, though practical, wide-spread, real-life applications may be a few years away.

Over the last few decades, DNA-focused gene therapy has become a reality. Now, instead of attacking just the symptoms of disease (surgically removing cancerous tumors or injecting insulin), doctors can actually tackle some deadly diseases and debilitating conditions by going right to the source to replace badly working versions of genetic material with shiny new ones.

However, currently available and approved DNA-focused drugs are few and far between. Researchers now believe that expanding the focus of gene therapy to RNA-focused drugs may allow researchers to reach further into our cellular library and drug many disease-related proteins currently considered undruggable.

At Los Alamos, work undertaken by Team Sanbonmatsu is helping to illuminate the hidden world of RNA, specifically long, non-coding RNA, and offers researchers a glimpse into the molecule’s structure, an essential step towards creating RNA-focused therapeutics.

Learn more on the Sanbonmatsu Team website.

Los Alamos news: Scientists image heart RNA structure for the first time