The 2024 Nobel Prize in Medicine Honors the Groundbreaking Discovery of MicroRNA

The Nobel assembly at the Karolinska Institutet in Stockholm, Sweden, has revealed the laureates of the 2024 Nobel Prize in Physiology or Medicine. Scientists Victor Ambros and Gary Ruvkun have been honored with the award for their discovery of microRNA and its significant role in post-transcriptional gene regulation. The two scientists will divide a total prize amounting to 11 million Swedish kronor, which converts to approximately one million US dollars.
MicroRNA consists of short RNA molecules that are essential for gene regulation, serving a vital function in an organism’s development and operations. These diminutive molecules can modulate the expression of numerous genes simultaneously, effectively coordinating and regulating entire gene networks.
Mutations that impact microRNA can lead to various health issues, including cancer, congenital hearing loss, skeletal disorders, and vision impairments. Currently, biopharmaceutical companies like Regulus Therapeutics are utilizing findings from microRNA research to create innovative drug modalities aimed at addressing numerous medical conditions.
A notable breakthrough
In the late 1980s, Victor Ambros and Gary Ruvkun collaborated as postdoctoral fellows at MIT within Robert Horvitz’s laboratory. Horvitz would go on to receive the Nobel Prize in 2002 for his contributions to understanding genetic regulation, alongside Sydney Brenner and John Sulston.
Their research efforts on genetics continued as Ambros established his own lab at Harvard University, which culminated in the publication of their discovery of the first microRNA molecule in two articles featured in the journal Cell in 1993. Since that time, it has become clear that microRNA-mediated gene regulation is a widespread phenomenon in multicellular organisms, with over a thousand human genes for microRNAs identified to date.
The revelation of microRNAs introduced a novel mechanism for post-transcriptional genetic regulation, affecting gene expression at the RNA level rather than through DNA manipulations. The action of microRNAs enables precise tuning of genetic expression, controlling the activation and deactivation of more than 60% of the protein-coding genes present in the human genome.
Bestowing the Nobel Prize upon the discovery of microRNA underscores the significance of genetic research in comprehending human physiology and the advancement of new therapeutic strategies to meet the pressing needs across a broad spectrum of diseases.