Victor Ambros and Gary Ruvkun. Image: Ill. Niklas Elmehed © Nobel Prize Outreach
A study of a small roundworm in the late 1980s led Victor Ambros and Gary Ruvkun to make a huge breakthrough in gene regulation.
Two scientists have been awarded the 2024 Nobel Prize in Physiology or Medicine for their discovery of microRNAs and the role they play in gene regulation.
Victor Ambros and Gary Ruvkun were honoured today (7 October) for their groundbreaking work, which revealed a completely new principle in how gene activity is regulated in multicellular organisms, including humans.
The Nobel Committee said their surprising discovery revealed an entirely new dimension to gene regulation.
The molecule was first discovered in the late 1980s when Ambros and Ruvkun were postdoctoral fellows at the same lab, where they were studying a small roundworm, C elegans.
The two scientists were interested in genes that control the timing of activation of different genetic programmes, ensuring that various cell types develop at the right time.
They studied two mutant strains of worms that displayed defects in the timing of activation of genetic programmes during development. They wanted to identify the mutated genes and understand their function.
As both scientists examined one strain each and compared their findings, it led to the breakthrough discovery of a new principle of gene regulation, mediated by a previously unknown type of RNA, microRNA.
The results were published in 1993 but were largely ignored for appearing to be irrelevant to humans and other complex animals. However, in 2000, Ruvkun’s research group published their discovery of another microRNA, which was present throughout the animal kingdom.
Over the following years, hundreds of different microRNAs were identified. It is now known that the human genome codes for more than one thousand microRNAs.
Ongoing research shows microRNAs to be fundamentally important for how organisms develop and function. For example, abnormal regulation by microRNA can contribute to cancer, and mutations in genes coding for microRNAs have been found in humans, causing conditions such as congenital hearing loss, eye and skeletal disorders.
Last week, in an interview with SiliconRepublic.com, FutureNeuro director Prof David Henshall said microRNAs are like “our genome’s master controller, making sure that the right amount of protein is made at the right time and in the right place for each cell in the body”.
Speaking today, Henshall described the announcement as “fantastic news”.
“The discovery of microRNAs has paved the way for significant advancements in medicine,” he said.
“Here in Ireland, researchers are at the forefront of microRNA research, studying their role in human disease and exploring their potential as treatments or biomarkers.
“At FutureNeuro, microRNAs are a cornerstone of our research. One exciting project involves developing a microRNA-based drug to treat epilepsy, a testament to the far-reaching impact of this Nobel prize-winning discovery.”
Prof Janosch Heller, assistant professor in biomedical sciences at Dublin City University, praised the Nobel laureates on their award. “Their pioneering work into gene regulation by microRNAs paved the way for groundbreaking research into novel therapies for devastating diseases such as epilepsy but also opened our eyes to the wonderful machinery that is tightly controlling what is happening in our cells.”
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With additional reporting by Rebecca Graham
