An unexpected discovery concerning gene regulation has earned Victor Ambros from the University of Massachusetts Chan Medical School and Gary Ruvkun from Harvard Medical School the 2024 Nobel Prize in physiology or medicine. The duo’s research identified small RNA segments, known as microRNAs, which play a significant role in regulating protein production in the body. This discovery, originating from their work with a tiny worm, has provided crucial insights into biological processes linked to health and disease.

MicroRNA’s Role in Gene Regulation

MicroRNAs are tiny RNA molecules that help regulate gene expression by affecting the production of proteins. In this process, microRNAs latch onto messenger RNA (mRNA), which carries instructions from DNA to make proteins. By clinging to mRNA, microRNAs prevent the translation of those instructions, reducing the amount of protein produced. Instead of acting as an on/off switch, these molecules function more like dimmers, subtly reducing protein production.

Early Discoveries in Worms

Ambros and Ruvkun’s research began in Caenorhabditis elegans, a small, transparent worm. Their focus was on two genes, lin-4 and lin-14, which played a key role in the worm’s development. Ambros initially discovered a small RNA segment associated with the lin-4 gene. It turned out to be the first identified microRNA. Ruvkun later demonstrated that the lin-4 microRNA binds to the mRNA of the lin-14 gene, reducing the production of its corresponding protein.

Impact on Human Health

MicroRNAs were initially thought to be specific to worms, but subsequent research revealed they are present across the animal kingdom, including humans. This discovery has opened up new avenues of research into how these small RNAs impact human health, with potential applications in treating diseases like cancer, heart disease, and neurodegenerative conditions.