Discovery of microRNA Earns Nobel Prize for Massachusetts Researchers

Gary Ruvkun, professor of genetics at Harvard Medical School and an investigator at Massachusetts General Hospital, and Victor Ambros, of the University of Massachusetts Chan Medical School. have received the 2024 Nobel Prize in Medicine for the discovery of microRNAs, a class of tiny RNA molecules that regulate the activities of genes in plants and animals, including humans.

Ruvkun and Ambros discovered the first microRNAs in animals and demonstrated how microRNAs can turn off genes whose activities are crucial for development. The two researchers’ discoveries revealed an entirely novel mechanism of gene regulation. Indeed, microRNAs are proving to be fundamentally important for how organisms develop and function, the Nobel committee said in its citation.

The code of life is stored in DNA, which is tightly guarded inside the cell nucleus. During cell division, DNA instructions from active genes get copied and carried outside the nucleus by messenger RNA into parts of the cell where they are translated into functioning proteins.

In the 1990s, Ruvkun’s and Ambros’ discoveries added a new twist to this classic plot. They identified thus-far unknown characters — microRNAs — that play a critical role, binding to specific messenger RNAs and shutting them down, thereby regulating which genes get translated into proteins and which genes get suppressed. By doing so, these tiny molecules can alter how organisms develop, mature, and function and malfunction.

The scientists’ work revealed that microRNAs are pivotal regulators of normal development and physiology of animals and plants as well as key players in an array of human diseases, including coronary heart disease, neurodegenerative conditions, and many forms of cancer.

miRNAs are now used in the clinic to determine tumor types and are implicated in heart disease, viral pathogenesis, regulation of neural function and disease, and the transition from so-called totipotent stem cells to differentiated cells. Human therapies based on miRNA regulation are already in clinical trials for heart disease. In plants, miRNAs mediate a variety of developmental and physiological transitions and turn out to have been key players in the actual domestication of corn, for example. (Source: Harvard Medical School Website, 07 October, 2024)