MicroRNA discovery earns 2024 Nobel Prize in Medicine: How this breakthrough in gene regulation benefits us

This year’s Nobel Prize in Medicine was awarded to Victor Ambros and Gary Ruvkun, as announced by the Nobel Assembly at the Karolinska Institute in Stockholm on October 7.  

Gary Ruvkun, professor of genetics at Harvard Medical School and an investigator at Massachusetts General Hospital, received the 2024 Nobel Prize in Physiology or Medicine for the discovery of microRNAs, a class of tiny RNA molecules that regulate the activities of genes in plants and animals, including humans.

Ruvkun shares the prize with his collaborator Victor Ambros from the University of Massachusetts Chan Medical School. Ambros and Ruvkun discovered the first microRNAs in animals and demonstrated how microRNAs can turn off genes whose activities are crucial for development.

They discovered microRNA, a new class of tiny RNA molecules that play a crucial role in gene regulation, according to an official statement.  

Their groundbreaking discovery revealed a completely new principle of gene regulation that turned out to be essential for multicellular organisms, including humans. It is now known that the human genome codes for over one thousand microRNAs. 

What is microRNA? 

Every cell in the human body contains the same DNA instructions, but different cells develop into specialised types — some become brain cells while others transform into muscle cells. 

The process by which cells decide their specific roles is guided by gene regulation, which directs which parts of the DNA are activated. 

Ribonucleic acid (RNA) typically functions as a messenger, carrying instructions from DNA to proteins — the essential building blocks that define a cell’s identity, whether it be brain or muscle. 

For example, messenger RNA (mRNA) vaccines developed during the COVID-19 pandemic work by delivering instructions to produce proteins that can combat viruses. 

Ambros and Ruvkun uncovered a new category of gene regulators that had previously gone unnoticed. Unlike traditional RNA, which transmits information, microRNA functions as a switch, turning genes on and off. 

Benoit Ballester, a French researcher, said, “The discovery of microRNAs introduced an additional layer of complexity, revealing that regions once considered non-coding are integral to gene regulation.” 

How this helps us? 

Numerous new treatments and tests involving microRNA are currently in trials, but none have been widely adopted yet. 

“While there are no clear applications for microRNAs at this moment, understanding their existence and their counter-regulatory networks is an essential first step,” Gunilla Karlsson Hedestam from the Karolinska Institute said during a press briefing in Stockholm. 

MicroRNAs hold particular promise in cancer treatment. Some of these molecular switches act as tumor suppressors, effectively putting a brake on inappropriate cell division, while others can promote cell division, potentially leading to cancer. 

Additionally, because many viruses utilise microRNAs, several antiviral drugs are in various stages of development, including treatments for hepatitis C. One challenge has been the instability of microRNAs, complicating their use in therapies. However, researchers are optimistic about using them as biomarkers to help identify specific types of cancer in patients. 

There is also emerging evidence that microRNAs could play a role in human evolution. Miska said, “It seems highly likely that microRNAs are significant in explaining the differences between the human brain and the brains of other primates.”