By Temitayo Ayetoto-Oladehinde
Victor Ambros and Gary Ruvkun, two American scientists won the Nobel Prize in physiology or medicine on Monday for their discovery of microRNA, tiny bits of genetic material that serve as on-and-off switches inside cells that help control what the cells do and when they do it.
Their work “prove to be fundamentally important for how organisms develop and function,” according to a panel that awarded the prize in Stockholm.
If scientists can better understand how they work and how to manipulate them, it could one day lead to powerful treatments for diseases like cancer.
Ambros and Ruvkun were initially interested in genes that control the timing of different genetic developments, ensuring that cell types develop at the right time, according to the Associated Press.
Their discovery ultimately “revealed a new dimension to gene regulation, essential for all complex life forms,” the panel said.
RNA is best known for carrying instructions for how to make proteins from DNA in the nucleus of the cell to tiny cellular factories that build the proteins. MicroRNA does not make proteins but helps to control what cells are doing, including switching on-and-off critical genes that make proteins.
Ambros and Ruvkun studied two mutant strains of worms commonly used as research models. The scientists set out to identify the mutated genes responsible for cell development in these worms and what their role was.
The mechanism they ultimately identified — the regulation of genes by microRNA — has allowed organisms to evolve for hundreds of millions of years.
“Their groundbreaking discovery revealed a completely new principle of gene regulation that turned out to be essential for multicellular organisms, including humans,” according to the citation explaining the importance of their work.
Ambros, currently a professor of natural science at the University of Massachusetts Medical School, performed the research at Harvard University. Ruvkun’s research was performed at Massachusetts General Hospital and Harvard Medical School, where he’s a professor of genetics.
The study of microRNA has opened up approaches to treating diseases like cancer because it helps regulate how genes work in our cells, said Claire Fletcher, a lecturer in molecular oncology at Imperial College London.
Fletcher said there were two main areas where microRNA could be helpful: in developing drugs to treat diseases and in serving as possible indicators of diseases, by tracking microRNA levels in the body.
“If we take the example of cancer, we’ll have a particular gene working overtime, it might be mutated and working in overdrive,” said Fletcher. She said scientists might one day be able to use microRNA to stop such effects.
Eric Miska, a geneticist at Cambridge University, said the discovery by Ambros and Ruvkun came as a complete surprise, overturning what scientists had long understood about how cells work.
“It was just a shock that there’s this whole new class of gene,” he said. He said the human genome has at least 800 microRNAs that are critical to how cells function.
Miska said there is ongoing work on the role of microRNA in infectious diseases like hepatitis and that it might also help treat neurological diseases.
Fletcher said studies are ongoing to see how microRNA approaches might help treat skin cancer, but no drugs have yet been approved. She expected that might happen in the next five to 10 years, adding that most treatments at the moment target cell proteins.
“If we can intervene at the microRNA level, it opens up a whole new way of us developing medicines,” she said.
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