The 2024 Nobel prize in physiology or medicine has been awarded jointly to two US-based scientists for the discovery of microRNAs – a class of tiny RNA molecules, around 22 base pairs long, that play a crucial role in gene regulation.
The Nobel committee awarded the prize to Victor Ambros from the University of Massachusetts’ Chan Medical School and Gary Ruvkun from Harvard Medical School ‘for the discovery of a fundamental principle governing how gene activity is regulated’. Ambros’ and Ruvkun’s findings help to explain how different cell types are produced from the same set of genetic instructions.
In the 1980s, Ambros and Ruvkun both worked as postdocs in the laboratory of Nobel laureate Robert Horvitz at the Massachusetts Institute of Technology, where they began studying genetic processes in the 1mm-long roundworm, Caenorhabditis elegans. The researchers continued this research after setting up their own independent laboratories. The pair’s complementary findings would eventually show how tiny microRNA molecules produced by one of the worm’s genes can bind to specific sites on mRNA molecules produced by another gene and block the production of the protein that the mRNA would otherwise code for.
Ambros and Ruvkun’s findings, published in two papers in Cell in 1993, revealed a previously unknown layer of gene regulation where microRNA could fine-tune protein production by targeting mRNA. MicroRNAs are now known to encompass a large group of regulators that control vast networks of protein coding genes. More crucially, this post-transcriptional regulatory mechanism has since been found across diverse species, with critical roles in animal development and adult tissue function.
Speaking at the announcement, the Nobel committee’s chair Gunilla Karlsson Hedestam said that the research ‘helps our basic understanding of how cells differentiate and become specialised’. She added that ongoing research based on the knowledge of microRNAs and their regulatory networks could one day lead to new treatments for some of the most challenging diseases. However, she highlighted the fundamental nature of the discovery, noting that it was ‘the first step towards developing applications’.
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