The Nobel assembly at Karolinska Institutet in Stockholm, Sweden, has announced the winners of the 2024 Nobel Prize in physiology or medicine. Scientists Victor Ambros and Gary Ruvkun have been awarded the prize “for the discovery of microRNA and its role in post-transcriptional gene regulation.” The two winners will share a prize of 11 million Swedish kronor, which is equivalent to about one million US dollars.
MicroRNA is a type of short RNA molecules that play a crucial role in gene regulation and are necessary for organisms to develop and function. These tiny molecules are able to regulate the expression of multiple different genes, coordinating and fine-tuning entire networks of genes.
Mutations affecting microRNA can contribute to a large number of diseases, including cancer, congenital hearing loss, skeletal disorders and sight impairments. Nowadays, biopharmaceutical companies such as Regulus Therapeutics are leveraging research on microRNA biology to develop new drug modalities against a wide range of conditions.
A groundbreaking discovery
In the late 1980s, Victor Ambros and Gary Ruvkun worked together as postdoctoral fellows at the Massachusetts Institute of Technology (MIT) in the laboratory of Robert Horvitz, who was later awarded the Nobel prize in 2002 for his research on genetic regulation mechanisms, alongside Sydney Brenner and John Sulston.
Their joint research on genetics continued as Ambros established his own laboratory at Harvard University, culminating with their discovery of the first microRNA molecule being published in two articles in the journal Cell in 1993. Since then, it has been established that gene regulation mediated by microRNA is an universal phenomenon in multicellular organisms, and more than a thousand genes for microRNAs have been identified in humans.
The discovery of microRNAs unveiled an entirely new mechanism of post-transcriptional genetic regulation, which refers to modifications to gene expression at the RNA level rather than the DNA level. The mechanism of action of microRNA allows for fine-tuning genetic expression, orchestrating the activation and deactivation of more than 60% of genes in the human genome that encode proteins.
Awarding the Nobel prize to the discovery of microRNA highlights the importance of genetic research for the understanding of human physiology and the development of new therapeutic modalities that can target unmet needs across a wide range of diseases.
