Victor Ambros and Gary Ruvkun have won the 2024 Nobel Prize in Physiology or Medicine for the discovery of microRNA and its role in gene regulation.
Victor Ambros is a Professor of Natural Science at the University of Massachusetts Medical School. Gary Ruvkun is a Professor of Genetics at Harvard Medical School.
Gene regulation ensures that only the correct set of genes is active in each specific cell type and that cellular functions keep adapting to changing conditions in our bodies and environment.
Ambros and Ruvkun discovered microRNA, tiny RNA molecules that play a crucial role in gene regulation. "Their groundbreaking discovery revealed a completely new principle of gene regulation that turned out to be essential for multicellular organisms, including humans," notes the Nobel Foundation in a press release.
In the late 1980s, Ambros and Ruvkun studied C. elegans worms. In particular, they investigated two mutant genes called lin-4 and lin-14, which resulted in anomalies in the activation of genetic programs during development.
Eventually, the two scientists discovered a new principle of gene regulation mediated by microRNA, a previously unknown type of RNA, and published their findings in 1993.
The research results, which seemed limited to C. elegans worms, weren't considered too significant after publication. But then, in 2000 Ruvkun’s research group discovered another mutant gene, the let-7 gene, which is present throughout the animal kingdom.
Gene regulation by microRNA is universal
In the following years, hundreds of different microRNAs were identified. Today, we know that there are more than a thousand genes for different microRNAs in humans, and that gene regulation by microRNA is universal among multicellular organisms.
"As the field exploded, which is just a joy to watch, then there was a sense that this is the sort of field, the sort of sea-change that gets awards and things," said Ruvkun in a first interview. "But that took a long time and was an unbelievable pleasure to watch, to participate in."
Advances are being made in developing microRNA-based diagnostics and therapeutics for metabolic disorders, cardiovascular diseases, neurodegenerative conditions, and cancer.
More detailed explanations are given in a document titled "Scientific background: For the discovery of microRNA and its role in post-transcriptional gene regulation."