Victor Ambros and Gary Ruvkun share 2024 Nobel Prize for Physiology or Medicine

The 2024 Nobel Prize for Physiology or Medicine has been jointly awarded to Victor Ambros, now at the University of Massachusetts Medical School, and Gary Ruvkun of Harvard Medical School for the discovery of microRNA (miRNA) for use in gene regulation.

The Nobel Assembly of Sweden’s Karolinska Institutet medical university made the announcement today, stating that the “surprising discovery revealed an entirely new dimension to gene regulation. MicroRNAs are proving to be fundamentally important for how organisms develop and function.”

Ambros was part of the team first discovered miRNAs in 1993 alongside Rosalind Lee, and Rhonda Feinbaum. While studying the development of the C. elegans roundworm, they identified lin-4 – a small RNA molecule – as a key regulator of developmental timing. Seven years later, Ruvkun was part of the team that discovered a second miRNA, let-7, which led to the realization that miRNAs are fundamental across species, including humans.

MiRNAs work by binding to complementary sequences in messenger RNAs (mRNAs), leading to the degradation or inhibition of the mRNA, thereby controlling protein synthesis.

Since their discovery, miRNAs have been found to be involved in a wide range of biological processes, including cell proliferation, differentiation, and apoptosis. They are also implicated in various diseases, especially cancer, cardiovascular disorders, and neurodegenerative diseases. miRNAs can function as oncogenes or tumor suppressors, depending on their target genes. This has spurred research into using miRNAs as biomarkers for disease diagnosis and prognosis, as well as potential therapeutic targets.

Today, miRNA-based therapies are being explored for conditions such as cancer and viral infections, with some candidates in clinical trials. Their ability to fine-tune gene expression continues to make miRNAs an exciting focus for therapeutic innovation.

The worlds of industry and science continue to anticipate the possibilities of miRNA technology now that it has developed and been elevated to the status of other Nobel Prize winning discoveries. The 2023 Nobel Prize was also jointly awarded to pharmaceutical scientists Katalin Karikó and Drew Weissman for the development of mRNA vaccines against COVID-19.