Victor Ambros and Gary Ruvkun honored for their discovery of microRNA and its pivotal role in post-transcriptional gene regulation, fundamentally changing our understanding of how genes are controlled.
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 groundbreaking discovery of microRNA and its fundamental role in post-transcriptional gene regulation.
This discovery, which began with studies of the tiny roundworm C. elegans in the 1990s, has revolutionized our understanding of how genes are controlled in cells. MicroRNAs are small RNA molecules that act as master regulators of gene expression, influencing virtually every biological process in multicellular organisms.
Dr. Victor Ambros, currently at the University of Massachusetts Chan Medical School, first identified the lin-4 gene in C. elegans and discovered it produced a small RNA rather than a protein. Working independently, Dr. Gary Ruvkun at Massachusetts General Hospital and Harvard Medical School demonstrated that this small RNA could regulate another gene, lin-14, by binding to its messenger RNA.
The implications of this discovery have been profound. Scientists have since identified over 1,000 different microRNAs in humans, each capable of regulating hundreds of different genes. Dysregulation of microRNAs has been linked to cancer, heart disease, neurological disorders, and autoimmune conditions.
Pharmaceutical companies are now developing microRNA-based therapeutics, with several candidates in clinical trials for conditions including hepatitis C, cancer, and cardiovascular disease. The field of microRNA diagnostics has also emerged, with blood-based microRNA panels being investigated as biomarkers for early disease detection.
"This discovery opened an entirely new dimension of gene regulation," said the Nobel Committee. "It has profoundly influenced our understanding of how organisms develop and function, and has opened new avenues for understanding and treating disease."
Both laureates will share the prize of 11 million Swedish kronor, continuing a legacy of recognizing fundamental discoveries that transform medicine.