Victor Ambros and Gary Ruvkun Awarded 2024 Nobel Prize for Groundbreaking MicroRNA Discovery

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Karolinska Institutet — The Nobel Assembly at the Karolinska Institutet has awarded the 2024 Nobel Prize in Physiology or Medicine to Victor Ambros and Gary Ruvkun for their groundbreaking discovery of microRNA and its pivotal role in post-transcriptional gene regulation. This significant achievement underscores a fundamental principle in understanding how gene activity is regulated in multicellular organisms, including humans.

Ambros and Ruvkun’s research focuses on the remarkable question of how diverse cell types—such as muscle and nerve cells—arise from identical genetic information stored within chromosomes. Their discovery of microRNAs, a class of small RNA molecules, has unveiled a new layer of gene regulation, crucial for the correct expression of genes in various cell types. It is now recognized that the human genome encodes over a thousand microRNAs, revealing a vital mechanism for cellular function and development.

The Nobel Prize specifically highlights how microRNAs regulate gene activity, which flows from DNA to messenger RNA (mRNA) and ultimately to protein production. This regulation is essential, as it ensures that only the appropriate genes are activated in specific cell types, enabling organs and tissues to function correctly. Aberrant gene regulation can lead to serious diseases, including cancer and diabetes, making the understanding of these processes critical in biomedical research.

Ambros and Ruvkun began their groundbreaking work in the late 1980s while postdoctoral fellows in Robert Horvitz’s laboratory, who won the Nobel Prize in 2002. Their investigations centered on the tiny roundworm *C. elegans*, a model organism well-suited for studying developmental processes. They examined mutant strains of the worm that exhibited timing defects in gene activation. Through their research, they discovered that the *lin-4* gene produced a short RNA molecule that inhibited the *lin-14* gene, establishing a new understanding of gene regulation.

Their findings, published in 1993, initially garnered little attention, perceived as an anomaly specific to *C. elegans*. However, in 2000, the identification of another microRNA, let-7, which is highly conserved across species, sparked significant interest. This led to the discovery of numerous microRNAs in humans and a universal understanding of microRNA regulation across multicellular organisms.

MicroRNA regulation has profound implications in various biological processes, influencing everything from development to disease progression. Disruptions in microRNA function have been linked to various conditions, including cancer and genetic disorders. Ambros and Ruvkun’s work has reshaped our understanding of gene regulation and opened new avenues for research in genetics and medicine.

Victor Ambros, born in 1953 in Hanover, New Hampshire, received his PhD from MIT and has held positions at Harvard University and Dartmouth Medical School. Gary Ruvkun, born in 1952 in Berkeley, California, obtained his PhD from Harvard and is a Professor of Genetics at Massachusetts General Hospital and Harvard Medical School.

The Nobel Prize in Physiology or Medicine, awarded since 1901, recognizes the most significant scientific discoveries that benefit humanity. Ambros and Ruvkun’s groundbreaking work exemplifies this mission, marking a pivotal moment in the field of molecular biology.

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