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Long-awaited Nobel Prize in medicine for gene regulation

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The discovery of microRNA being awarded a Nobel Prize is something many within the field have anticipated for a long time. This is the view of researchers at the University of Gothenburg. The Nobel Prize in Physiology or Medicine highlights a fundamental principle of how gene activity is regulated.

This year's Nobel Prize in Physiology or Medicine has been awarded to researchers Victor Ambros and Gary Ruvkun, both based in the USA, for their discovery of microRNA and its role in what is called post-transcriptional gene regulation.

Ka-Wei Tang, Associate Professor of Clinical Virology at Sahlgrenska Academy, University of Gothenburg, explains:

“A human being consists of 30 trillion cells. Each cell contains exactly the same instruction manual, DNA, which holds the information on how to build proteins to form muscle cells, nerve cells, and so on. To create protein, the instructions from DNA are copied to mRNA, which then serves as a template for building proteins," he says, and continues:

“Since each cell contains the same information, the cells must regulate which proteins are expressed in the different cell types. Regulation occurs at the DNA level, but Ambros and Ruvkun showed that regulation can also happen at the mRNA level. They discovered that small RNA, microRNA, regulates the amount of mRNA and thereby affects how much protein is expressed in the cell."

Drugs in development

“It turns out that this regulation occurs in most living organisms. Diseases like cancer, for example, can arise when mutations occur in these microRNAs. Currently, there are no microRNA-based drugs, but several are under development," says Ka-Wei Tang.

Marc Pilon, Professor of Genetics at the Faculty of Science, University of Gothenburg, shares insights into the journey leading to the award-winning discovery, which began with studies of mutants in the roundworm Caenorhabditis elegans, abbreviated C. elegans, a model organism frequently used in molecular biology research.

The laureates discovered that microRNA, a class of tiny RNA molecules, plays a crucial role in gene regulation. This groundbreaking discovery revealed a new mechanism for regulating genes, which turned out to be central to multicellular organisms.

A new principle for gene regulation

“We now know that many of these microRNAs are encoded in the genomes of animals, with well over 2,000 of them in the human genome, where they regulate the majority of protein-coding genes, using the same mechanisms that were originally discovered in C. elegans,” says Marc Pilon.

“Victor and Gary are both fantastic scientists who passionately pursue their scientific questions wherever they lead, in this case to a new principle for gene regulation that is evolutionarily conserved in all animals,” says Marc Pilon.

Erik Lekholm, Professor of Bioinformatics at the Sahlgrenska Academy, University of Gothenburg, specializing in large-scale analysis of DNA and RNA, adds:

“This is a prize that has long been speculated about and that many in the microRNA field have been eagerly awaiting. The discovery adds a new layer to gene regulation, the process that allows cells to look different and perform different roles in the body, despite all containing the same DNA. MicroRNA genes have been shown to play roles in virtually every conceivable cellular process, and today they have, among other things, diagnostic applications. Above all, it's about very important fundamental cell biology."

Adjunct Senior Lecturer Ka-Wei Tang, Institute of Biomedicine, Professor Marc Pilon, Department of Chemistry and Molecular Biology and Professor Erik Lekholm, Institute of Biomedicine.<br /> Photo: Lisa Thanner, Göteborgs universitet, Elin Lindström
Photo: Lisa Thanner, Göteborgs universitet, Elin Lindström