The 2024 Nobel Prize in Physiology or Medicine has been awarded to American scientists Victor Ambros and Gary Ruvkun for their groundbreaking discovery of microRNA (or miRNA) and its role in post-transcriptional gene regulation.
In a statement, the Nobel Assembly said the laureates discovered a new class of non-coding RNA molecules that play a crucial role in gene regulation.
Their breakthrough revealed a previously unknown mechanism by which genes are controlled that is essential for the development and function of multicellular organisms, including humans.
A gene contains instructions within our DNA. To activate these instructions, cells create a copy called messenger RNA (mRNA), which exits the nucleus and directs the cell’s machinery to produce a specific protein.
However, microRNAs can bind to this mRNA, preventing it from functioning. Essentially, microRNAs block the expression of the gene by stopping the production of the corresponding protein from mRNA’s instructions, effectively regulating which genes are “turned on” or “off” in the cell. This control plays a critical role in development and cellular function.
Today, it’s known that the human genome encodes over a thousand microRNAs, which play vital roles in how organisms grow, differentiate and operate, uncovering a new layer of complexity in genetic regulation.
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The duo’s research explained how cells with identical genetic material can develop into specialized types, such as muscle and nerve cells. Despite containing the same set of genes, cells are able to differentiate due to the regulatory role of microRNAs, which control gene expression in a precise manner. This discovery helped illuminate the mechanisms behind cellular specialization and development, revealing how genes are selectively activated or silenced in different tissues to ensure proper growth and function.
Ruvkun told Reuters that the Nobel Prize is akin to ‘The Show’ in Major League Baseball. “Which means it’s not any show, it’s the show,” he said.
Speaking on his collaboration with Ambros and receiving previous awards, he said the duo had been “joined at the hip for quite a while.”
Ruvkun said “That’s been great. He’s a wonderful guy.” Ambros echoed his sentiments saying he was happy to share the award with “a great friend.”
Professor Ambros works at the University of Massachusetts Medical School, and Professor Ruvkun at Harvard Medical School.
Ambros and Ruvkun made the discovery as they were exploring how different cell types develop, leading to their identification of microRNAs — a new class of small RNA molecules critical to gene regulation.
The work, which began in the early 1990s through studies on the C. elegans roundworm, revealed that microRNAs play a critical role in controlling the activity of genes post-transcriptionally.
Through this, they discovered the first microRNA, lin-4, and its role in controlling the expression of other genes, particularly lin-14, through complementary base pairing with its mRNA’s 3’ untranslated region (UTR). This regulatory mechanism opened new insights into post-transcriptional gene regulation, revealing a complex and conserved biological process.
They showed that mutations in lin-4 disrupted proper developmental timing, suggesting that miRNAs were essential in coordinating stages of development. These findings were complemented by the identification of another miRNA, let-7, in 2000 by the Ruvkun lab, which was conserved across species and further confirmed miRNAs’ roles in regulating genes critical to developmental timing and cell differentiation.
The initial discovery of lin-4 in 1993 marked a significant departure from the prevailing understanding of gene regulation, which was focused primarily on transcriptional controls.
Ambros and Ruvkun’s work demonstrated that small RNAs could repress gene expression after mRNA was made, pointing to a previously unknown layer of genetic control.
Their findings have profound implications for understanding developmental biology, cancer and other diseases, as microRNAs are crucial regulators of cellular processes across many organisms.
The winners will split a prize fund of 11 million Swedish kronor ($1.1 million).
In addition to their direct applications in understanding human health and disease, the discovery of microRNAs has spurred advancements in biotechnology and medicine.
How MicroRNA Therapeutics Work
MicroRNA-based diagnostics and therapeutics are in development for various conditions, as they have emerged as promising biomarkers for disease states and therapeutic targets. Their ability to modulate gene expression at the RNA level provides a novel therapeutic approach for conditions.
For example, microRNA research has paved the way for new approaches to treating diseases like cancer by regulating how genes function in cells, Dr. Claire Fletcher, a molecular oncology expert at Imperial College London, told AP News.
There are two key areas where microRNAs show promise: drug development and disease diagnosis, particularly through tracking miRNA levels as biomarkers.
In cancer, for instance, overactive genes could be controlled and downregulated with microRNA-based therapies.
Once in the shadows of DNA, interest in RNA has been increasing within both scientific and public circles. Last year’s Nobel Prize in Physiology and Medicine also went to RNA researchers. Hungarian scientist Katalin Kariko and US colleague Drew Weissman shared the 2023 prize for developing mRNA technology that led to the mRNA COVID-19 vaccines.
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