Victor Ambros and Gary Ruvkun received the 2024 Nobel Prize for their groundbreaking discovery of microRNA and its role in post-transcriptional gene regulation, opening new therapeutic avenues.
The Nobel Assembly at Karolinska Institutet has awarded the 2024 Nobel Prize in Physiology or Medicine jointly to Victor Ambros and Gary Ruvkun for their discovery of microRNA and its role in post-transcriptional gene regulation.
This revolutionary finding has fundamentally changed our understanding of how genes are regulated in cells and organisms. MicroRNAs are tiny RNA molecules, typically only 20-24 nucleotides long, that act as master regulators of gene expression by binding to messenger RNA and either degrading it or preventing its translation into protein.
The discovery began in the early 1990s when both scientists were studying the development of the nematode worm C. elegans. Ambros, working at Harvard University, identified an unusual gene called lin-4 that controlled developmental timing but did not encode a protein. Instead, it produced a small RNA molecule.
Meanwhile, Ruvkun at Massachusetts General Hospital discovered that lin-4 regulated another gene, lin-14, by binding to a specific sequence in its messenger RNA. This was the first demonstration of what would become known as microRNA-mediated gene silencing.
For years, many scientists believed this mechanism was a quirk specific to worms. The pivotal moment came in 2000 when Ruvkun's team discovered let-7, a microRNA conserved across animal species including humans. This proved that microRNA regulation was a fundamental biological mechanism.
Today, over 2,500 human microRNAs have been identified, and they are estimated to regulate more than 60% of all protein-coding genes. Dysregulation of microRNAs has been implicated in numerous diseases including cancer, cardiovascular disease, diabetes, and neurological disorders.
The therapeutic potential is enormous. Several companies are developing microRNA-based drugs, with candidates in clinical trials for liver disease, cancer, and cardiovascular conditions. MicroRNA expression profiles are also being used as diagnostic biomarkers for disease detection and prognosis.
Both laureates expressed gratitude to the scientific community and their mentors. Ambros credited his time studying with Nobel laureate David Baltimore, while Ruvkun acknowledged the importance of the collaborative C. elegans research community.
The prize of 11 million Swedish kronor will be shared equally between the two scientists.