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Wara Samar

9th October 2024

The Tiny RNA Revolution: Nobel Prize 2024 for Discovery of microRNA in Gene Control

The 2024 Nobel Prize in Physiology or Medicine honors two scientists, Victor Ambros and Gary Ruvkun, for their discovery of microRNAs and their important role in gene regulation. Their work has revealed a fundamental mechanism that helps determine which genes are activated in our cells, shaping the development and function of multicellular organisms, including humans.

How Cells Decide Which Genes to Activate

Every cell in our body contains identical genetic material stored in DNA. Yet, different types of cells, such as nerve and muscle cells, perform entirely distinct functions. How does this happen? The answer lies in the precise regulation of gene activity. This fine-tuned control ensures that the right genes are turned on in the right cells at the right time, allowing for the proper development and functioning of tissues and organs.

Ambros and Ruvkun’s discovery of microRNAs—a new class of tiny RNA molecules—provided a key to understanding this regulation. MicroRNAs don’t code for proteins but instead play a vital role in controlling the production of proteins by targeting messenger RNAs (mRNAs). This discovery, which was initially met with skepticism, has since revolutionized our understanding of gene expression.

The Surprising Discovery in a Tiny Worm

The journey to this discovery began in the lab of Robert Horvitz, a Nobel laureate himself, where both Ambros and Ruvkun worked in the late 1980s. They were studying a tiny roundworm called Caenorhabditis elegans, a favorite model organism in genetics research. Despite its small size, C. elegans has specialized cells similar to those found in larger animals, making it ideal for studying development.

Robert Horvitz

Ambros and Ruvkun were intrigued by mutant worms that showed developmental timing defects. Through their investigations, they discovered that a tiny RNA molecule, later identified as microRNA, was responsible for inhibiting the activity of specific genes. This finding was a game-changer in understanding how genes are regulated at the post-transcriptional level—after the mRNA is produced but before proteins are made.

A New Principle of Gene Regulation

Ambros and Ruvkun’s research demonstrated that microRNAs work by binding to complementary sequences in mRNAs, effectively blocking the production of proteins. This unexpected finding was published in 1993, but it took years for the scientific community to fully appreciate its significance.

Over time, further research showed that microRNA-mediated regulation is not unique to worms—it’s a universal mechanism found in all multicellular organisms. In fact, humans have more than a thousand genes that produce microRNAs, making them essential for different biological processes.

The Impact on Human Health

Gene regulation by microRNAs is now known to be crucial for the proper development and function of cells and tissues. When this regulation goes awry, it can lead to a variety of diseases, including cancer, diabetes, and autoimmune disorders. In some cases, mutations in microRNA genes have been linked to congenital conditions, such as hearing loss and skeletal disorders.

Ambros and Ruvkun’s discovery opened a new chapter in the study of genetics, offering insights into how complex life forms evolve and function. Their work continues to have profound implications for medicine, particularly in understanding and treating diseases related to gene regulation.

The Pioneers Behind the Discovery

Victor Ambros and Gary Ruvkun’s scientific journey has been nothing short of remarkable. Ambros, born in 1953, is currently the Silverman Professor of Natural Science at the University of Massachusetts Medical School. Ruvkun, born in 1952, is a Professor of Genetics at Harvard Medical School. Together, their discovery of microRNAs has reshaped the landscape of molecular biology, earning them a well-deserved place among the giants of modern science.

Their pioneering work highlights how tiny molecules can have monumental effects, not just in worms, but across the animal kingdom, including in humans. The discovery of microRNA has not only broadened our understanding of gene regulation but has also opened new avenues for treating diseases tied to genetic misregulation.

MedTech World extends its heartfelt congratulations to Victor Ambros and Gary Ruvkun for their remarkable achievement.

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