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US scientists Victor Ambros and Gary Ruvkun have won the medicine Nobel Prize for discovering tiny molecules that have played a crucial role in human evolution, offering clues to how deadly diseases can be tackled.
The two researchers will share SKr11mn ($1.06mn) for their discovery of micro ribonucleic acid (microRNA) and its involvement in gene regulation, the Nobel Assembly said on Monday as it announced the first of its six annual awards.
Genes are the units of heredity passed from one generation to the next that determine physical and biological characteristics. MicroRNA has proved to be hugely important to understanding how organisms develop and function normally — and what has gone wrong when they do not.
The Nobel Assembly in Stockholm lauded Ambros and Ruvkun’s discovery as “seminal” and “unexpected”, revealing “a new dimension to gene regulation, essential for all complex lifeforms”.
“Their pioneering work into gene regulation by microRNAs paved the way for groundbreaking research into novel therapies for devastating diseases such as epilepsy,” said Janosch Heller, assistant professor in biomedical Sciences at Dublin City University. “[They] also opened our eyes to the wonderful machinery that controls what is happening in our cells.”
The prize-winning pair’s discovery transformed understanding of gene regulation thought to have been settled in the 1960s. Researchers then had established a mechanism involving DNA, the bearer of genetic information, and messenger RNA (mRNA), which carries instructions to synthesise crucial proteins.
In this method, specialised proteins known as transcription factors bound themselves to specific regions in DNA, determining which types of mRNA were produced.
The Nobel Prize-winning breakthrough that revolutionised this model began with observations of a 1mm-long roundworm named Caenorhabditis elegans (C. elegans).
Ruvkun, born in 1952, and Ambros, born the following year, studied the creature as post-doctoral fellows in the late 1980s in the laboratory of Robert Horvitz, himself a Nobel Prize co-winner in 2002.
C. elegans is favoured by researchers because it contains within its tiny body specialist cell types such as nerves and muscles, making it a good model for bigger and more complex animals.
In the early 1990s, the researchers began finding results that appeared to show a new mechanism of gene regulation. Ambros, working at Harvard university, and Ruvkun, based at Massachusetts General Hospital and Harvard Medical School, realised they had discovered microRNA.
Further research has revealed that microRNA has been influencing gene regulation for hundreds of millions of years and has played a critical role in the evolution of complex organisms.
More than 1,000 types of microRNA have been identified and they are fundamental to the development of healthy cells and tissues. Abnormal functioning of microRNA can contribute to diseases from cancers to hearing loss, eye damage and skeletal disorders.
MicroRNA networks are observed to be disrupted in many tumours, offering avenues for research into potential cancer treatments.
Ambros is now a professor of natural science at the University of Massachusetts Medical School. Ruvkun is an investigator at Massachusetts General Hospital and Harvard Medical School.
The Nobel Prizes in physics, chemistry, literature, peace and economics will all be unveiled over the next week.
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