Victor Ambros from the University of Massachusetts Medical School and Gary Ruvkun from Harvard Medical School jointly received the 2024 Nobel Prize for their discovery of microRNA and its role in post-transcriptional gene regulation.
Also Read: First Full Map of Every Neuron in a Fruit Fly Brain
The discovery of microRNA introduced a new principle in gene regulation, overturning the previously understood model known as the central dogma of molecular biology, where RNA simply carries instructions from DNA to cells to create proteins.
Ambros and Ruvkun found that microRNAs regulate the activity of messenger RNA (mRNA), the RNA type responsible for instructing cells on protein synthesis.
These molecules ensure that proteins are produced in the right amounts at the right time. Disruptions in this balance can lead to various diseases such as cancer, osteoporosis, diabetes and even congenital disorders.
Research into microRNA has revealed that mutations in genes regulating microRNAs are linked to multiple diseases. These include congenital hearing loss, skeletal abnormalities and other developmental disorders.
microRNAs play a crucial role in cellular health by controlling gene expression, preventing the overproduction or underproduction of proteins, which can lead to malignant growths, immune dysfunctions or metabolic issues.
Current clinical trials are exploring treatments based on microRNAs to tackle diseases like cancer, heart disease and neurodegenerative conditions.
Both Victor Ambros and Gary Ruvkun started their careers at the Massachusetts Institute of Technology in the lab of H. Robert Horvitz, a Nobel Prize laureate himself in 2002.
Their interest in gene regulation and developmental timing in organisms began during their postdoctoral research and continued after they established their respective laboratories at Harvard Medical School and the University of Massachusetts Medical School.
In 2008, Ambros and Ruvkun along with David C. Baulcombe of the University of Cambridge were awarded the Albert Lasker Basic Medical Research Award for their research on microRNA.
The first scientific papers describing the role of microRNA in gene regulation published in 1993, were met with doubt, as many believed that microRNA’s regulatory function was unique to the C. elegans worm species, the organism used in their studies.
However, subsequent research confirmed that microRNA plays a role in thousands of gene expressions in humans and other complex organisms.
The Nobel Committee praised this discovery as a new dimension to gene regulation that is essential for all multicellular life forms, not just simple organisms like worms.
Also Read: Sub-Earth Exoplanet Barnard b Discovered Orbiting the Closest Star to the Solar System
Scientists now know that the human genome provides instructions for over 1,000 forms of microRNA, which influence a wide array of biological functions from cell differentiation to immune responses.
Clinical trials focusing on microRNA-based therapies are underway particularly for conditions like heart disease, cancer and skin disorders.
Experts predict that microRNA therapies may be introduced in the next 5 to 10 years. Dr. Claire Fletcher, a lecturer in molecular oncology at Imperial College London, said that microRNAs hold promise in treating diseases by either suppressing harmful gene activity or boosting protective genes.
microRNA (miRNA) refers to tiny RNA molecules that do not code for proteins. Instead, they play a crucial role in regulating gene expression by binding to messenger RNA (mRNA) and preventing the production of specific proteins.
miRNAs were thought to be an oddity limited to the small roundworm C. elegans, but later research showed that miRNAs are present across a variety of species including humans.
These small RNA molecules work by either blocking the mRNA from being read by the cell’s protein-making machinery or causing the mRNA to degrade, ensuring that the gene’s instructions are not carried out.
The role of microRNA in post-transcriptional gene regulation refers to its ability to influence gene expression after the DNA has already been transcribed into mRNA.
This process helps explain how cells with identical DNA can behave and function differently such as in the differentiation of muscle cells and nerve cells.
Ambros and Ruvkun’s research revealed that microRNA is a regulator in selecting which mRNAs are translated into proteins, controlling the cell’s protein production at a critical juncture.
The journey to uncover microRNA began in the early 1990s when both scientists worked with the small roundworm Caenorhabditis elegans (C. elegans). They found that certain tiny RNA molecules in C. elegans could bind to mRNA and regulate the production of proteins, something that was previously not understood.
Victor Ambros and Gary Ruvkun were each awarded a cash prize of 11 million Swedish kronor, a sum provided by a bequest from the Nobel Prize’s founder, Alfred Nobel.
The award recognizes not only their individual achievements but also the impact of basic scientific research on improving human health.
In a statement following the award announcement, Dr. Jon Lorsch, the director of the National Institute of General Medical Sciences addressed that the discovery of microRNA has provided new possibilities for treating previously untreatable diseases.
Also Read: China Unveils New Lunar Spacesuit for Manned Moon Mission by 2030
