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Nobel Prize

Two share Nobel Prize in Physiology or Medicine for microRNA discoveries

Victor Ambros and Gary Ruvkun receive prize for the discovery of microRNA and its role in post-transcriptional gene regulation

by Max Barnhart
October 10, 2024 | A version of this story appeared in Volume 102, Issue 32

 

Two smiling white men in tuxedos.
Credit: AP Photo/Steven Senne
Gary Ruvkun (left) and Victor Ambros

Victor Ambros from the University of Massachusetts Chan Medical School and Gary Ruvkun from Harvard Medical School have won the 2024 Nobel Prize in Physiology or Medicine. They share the prize for their discovery of microRNAs, a family of molecules produced in the body that can profoundly alter gene expression.

In a press conference, Ruvkun remarked on his career, saying that “the surprises are what keep you young in science.” He also said of the model organism Caenorhabditis elegans, in which he helped discover microRNAs, that their potent small RNA system “makes our little worms badass . . . and I was asserting this before the Nobel stinkin’ Prize.”

RNA biology is now the subject of a third Nobel Prize–winning discovery in the 21st century. Last year’s award in physiology or medicine went to Katalin Karikó and Drew Weissman for their work on messenger RNA (mRNA) vaccines, and in 2006, Andrew Fire and Craig Mello won the prize for their discovery of RNA interference.

“Honestly, this was not something that I expected,” Ambros said at a press conference. “In my opinion, the Nobel Prize to Mello and Fire encompassed all these phenomena that we studied.” He also thanked his wife, Rosalind “Candy” Lee for her crucial role in the discovery of microRNAs; Lee was a first author and Ambros last author on the Cell paper in which the production of a microRNA was first described (1993, DOI: 10.1016/0092-8674(93)90529-y).

An illustration of a small length of twisted RNA sticking onto a longer RNA length. To the right is a sketch of the nematode Caenorhabditis elegans.
Credit: Mattias Karlén/The Nobel Committee for Physiology or Medicine
Victor Ambros and Gary Ruvkun discovered microRNA while studying the worm Caenorhabditis elegans. Ambros found that the gene lin-4 encoded a tiny RNA that did not code for a protein, and when Ruvkun cloned the lin-4 gene, the two scientists realized that the lin-4 microRNA sequence (red) matched a complementary sequence in the lin-4 messenger RNA (blue).

MicroRNAs are short, single-stranded RNA molecules, one of several classes of non-protein coding RNAs that regulate gene expression. Scientists have been working toward unlocking the therapeutic potential of microRNAs, which can also serve as biomarkers for some diseases.

“It’s an RNA world we live in,” says Matthew Disney, a chemist at the University of Florida and scientific founder of Expansion Therapeutics, which is working on medicines that target microRNAs. “We’ve come through an era where RNA was the disease. SARS-CoV-2 has an RNA genome. And RNA is the cure: the mRNA vaccines.”

“I think this is going to further cement the importance of RNA,” he adds.

With additional reporting by Sarah Braner, Laura Howes, and Laurel Oldach

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