The 2024 Nobel Prize in Physiology or Medicine has been awarded to Victor Ambros and Gary Ruvkun for their discovery of microRNA.
“Gene regulation by microRNA, first revealed by Ambros and Ruvkun, has been at work for hundreds of millions of years. This mechanism has enabled the evolution of increasingly complex organisms,” said the Nobel Assembly at Karolinska Institutet, making the award.
“Ambros and Ruvkun’s seminal discovery in the small worm C. elegans was unexpected, and revealed a new dimension to gene regulation, essential for all complex life forms,” it said.
The discovery of microRNA, a class of tiny RNA molecules that play a crucial role in gene regulation, helped to solve a scientific puzzle. Every cell contains the same chromosomes, which means every cell has the same set of genes and the instruction set. But clearly not all cells are the same, and have their own characteristics and functions courtesy of gene regulation, which allows each cell to select only the relevant instructions.
As the Nobel Assembly explained, genetic information passes from DNA to messenger RNA (mRNA), via a process called transcription, and then on to the cellular machinery for protein production. There, mRNAs are translated so that proteins are made according to the genetic instructions stored in DNA.
Since the 1960s it has been known that specialized proteins known as transcription factors can bind to specific regions in DNA and control the flow of genetic information by determining which mRNAs are produced. Then, in 1993, Ambros and Ruvkun published their work on a new level of gene regulation based on research on the 1 mm long roundworm, C. elegans. This work demonstrated a new principle of gene regulation, mediated by a previously unknown type of RNA - microRNA.
While the results were considered interesting, was considered a peculiarity of this particular worm and not applicable to human biology. It was only in 2000 that when Ruvkun’s research group published their discovery of another microRNA, that interest in their discovery grew.
“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,” said the Nobel Assembly.
Ambros is now Silverman Professor of Natural Science at the University of Massachusetts Medical School. Ruvkun is Professor of Genetics at Harvard Medical School.
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