Nobel Prize in Medicine 2024 awarded to Victor Ambros and Gary Ruvkun for work on the discovery of microRNA

CNN
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This year's Nobel Prize in Physiology or Medicine was awarded to Victor Ambros and Gary Ruvkun for their work discovering microRNA, a fundamental principle that controls the regulation of gene activity.

Their research revealed how genes give rise to different cells in the human body, a process known as gene regulation.

Gene regulation by microRNA – a family of molecules that help cells control the type of proteins they make – has been at work for hundreds of millions of years, but was first discovered by Ambros and Ruvkun.

The Nobel Prize Committee announced the prestigious award, considered the pinnacle of scientific achievement, on Monday in Sweden.

It praised the “groundbreaking discovery” by American biologists, which, according to the committee, “revealed a completely new dimension of gene regulation.”

Ambros, a professor of natural sciences at the University of Massachusetts Medical School, conducted the research that earned him the prize at Harvard University. Ruvkun conducted his research at Massachusetts General Hospital and is a professor of genetics at Harvard Medical School.

“The information stored in our chromosomes can be compared to an instruction manual for all the cells in our body. Every cell contains the same chromosomes, so every cell contains exactly the same set of genes and exactly the same set of instructions,” the committee said in a statement detailing the duo’s work.

And yet different cell types – such as muscle and nerve cells – have different properties. The two biologists have spent their careers studying how these differences arise.

“The answer lies in gene regulation, which allows each cell to select only the relevant instructions. This ensures that only the correct set of genes is active in each cell type,” the committee said.

Gene regulation by microRNA has supported the development of increasingly complex organisms. When gene regulation fails, it can lead to cancer and other diseases that occur in humans and other animals, such as hearing loss and skeletal diseases.

In their early work, the pair examined the genetic makeup of a tiny, 1-millimeter-long roundworm, C. elegans. Despite its small size, this worm has many specialized cell types, such as nerve and muscle cells, that are also found in larger, more complex animals, making it a useful model for studying tissue development and maturation in multicellular organisms.

“The first microRNA was discovered by Victor Ambros in 1993, but for more than seven years it was thought to be a curiosity peculiar to a small worm, C. elegans,” said Olle Kämpe, professor of endocrinology at Karolinsa Institutet and Vice Chairman of the Nobel Medicine Committee.

That 1993 discovery was met with “deafening silence” and was initially considered irrelevant to humans, the committee said, until Ruvkun published his discovery of another microRNA found in the animal kingdom.

“Then the field exploded,” said Kämpe. “Today, more than tens of thousands of microRNAs have been identified in various organisms.”

Many have been waiting for Ambros and Ruvkun to receive the Nobel Prize for years, said David Pendlebury, head of research analysis at the Clarivate Institute for Scientific Information.

“They (microRNAs) offer potential diagnostic and therapeutic opportunities in the treatment of cancer and other diseases. Clinical trials are underway to use microRNA profiling for patient prognosis and clinical response,” Pendlebury told CNN.

Last year, the prize was awarded to Katalin Karikó and Drew Weissman for their work on MRNA vaccines, a crucial tool to curb the spread of Covid-19.

The prize is worth 11 million Swedish crowns (US$1 million).