This year’s Nobel Prize in physiology or medicine has been awarded to Victor Ambros and Gary Ruvkun. They were recognized for their discovery of microRNA (miRNA) molecules, which regulate gene expression primarily at the post-transcriptional level by influencing mRNA translation or stability. This makes miRNAs key players in numerous physiological and pathological processes.

Many researchers at IBCH PAS are involved in the study of miRNAs. At our institute, research is conducted, among others, on miRNA biogenesis, the role of miRNA in cancer and neurological diseases, studies of the structure of miRNAs and their precursors, research into mutations in miRNA-encoding genes and their impact on the function of these molecules, the development of artificial miRNAs for therapeutic use in specific diseases, as well as studies on the role of miRNAs in plants.

Prof. Piotr Kozłowski: “In the early 1990s, Victor Ambros and Gary Ruvkun, American scientists working in Boston, discovered a completely new principle of gene expression regulation while studying the development of the worm Caenorhabditis elegans. They observed that genes could be regulated by short, approximately 20-nucleotide-long non-coding RNA molecules, later named microRNAs (miRNAs). Their groundbreaking discoveries were described in two articles published in Cell in 1993 ^1,2.

According to the canonical miRNA mechanism, by complementarily interacting with specific mRNAs (their targets), miRNAs can block their translation, thereby negatively regulating gene expression. In the following years, miRNAs were identified in many other organisms, including plants, insects, and vertebrates, including humans. It has also been shown that they are involved in regulating many cellular processes. The human genome encodes over 1,000 different miRNAs, which are estimated to regulate the expression of most human genes. Dysregulation of miRNAs has been linked to numerous diseases, including neurological disorders and cancers. Currently, many miRNAs are being tested as potential therapeutics or targets for molecular therapies.”

Anna Kurzyńska-Kokorniak, PhD, DSc, Assoc. Prof.: “miRNA molecules are fundamental for the development and functioning of organisms. Since their discovery, research has been ongoing worldwide to determine the role of specific miRNAs in the organism (with respect to the type of cell, tissue, or organ) and to identify which diseases may be linked to the improper functioning of specific miRNAs. A particularly interesting case is identical miRNAs that arise from different precursor molecules, which, according to the latest research, may perform distinct functions within the cell.

miRNA molecules can also be found in body fluids: blood, urine, and breast milk. miRNAs released into the bloodstream, known as circulating miRNAs, may serve as diagnostic biomarkers. For example, profiling miRNA expression is helpful in diagnosing cardiovascular disorders, determining cancer types, monitoring the progression of cancer, and assessing the radicality of surgical procedures.

In therapies based on miRNA regulation, a particular challenge is the method and effectiveness of delivering therapeutic molecules to specific cells where they are needed, as well as ensuring the specificity and durability of the therapeutic effect.”

Recognizing the significance of today’s Nobel Prize for the advancement of science, we also encourage you to read Victor Ambros’s article on miRNA-mediated gene regulation, published earlier this year in Advances in Biochemistry, the journal whose Editor-in-Chief is Kamilla Grzywacz, PhD, DSc, Assoc. Prof., from the Laboratory of Invertebrate Model Organisms.

Link to the article: https://postepybiochemii.ptbioch.edu.pl/…/article/view/515

¹ Lee RC, Feinbaum RL, Ambros V. The C. elegans heterochronic gene lin-4 encodes small RNAs with antisense complementarity to lin-14. Cell. 1993;75(5):843-854. doi:10.1016/0092-8674(93)90529-y
² Wightman B, Ha I, Ruvkun G. Posttranscriptional regulation of the heterochronic gene lin-14 by lin-4 mediates temporal pattern formation in C. elegans. Cell. 1993;75(5):855-862. doi:10.1016/0092-8674(93)90530-4