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

The role of microRNAs in gene regulation

MicroRNAs are tiny RNA molecules that help regulate gene expression by influencing protein production. In this process, microRNAs attach to messenger RNA (mRNA), which carries instructions from DNA to make proteins. By attaching to mRNA, microRNAs prevent the translation of these instructions, reducing the amount of protein produced. Rather than acting as an on/off switch, these molecules function more as dampeners, subtly reducing protein production.

Early discoveries at Worms

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

Impact on human health

MicroRNAs were initially thought to be specific to worms, but later research revealed that they are present throughout the animal kingdom, including humans. The discovery opened new avenues of research into how these small RNAs affect human health, with potential applications in the treatment of diseases such as cancer, heart disease and neurodegenerative conditions.