2024-10-08 10:19:03
Will the discovery of microRNAs, which earned American scientists Victor Ambros and Gary Ruvkun the 2024 Nobel Prize for Medicine, have any effects on disease treatments? Or is there already an open path in the medical field? What effects are triggered by the discoveries?
“The implications are of two types, both scientific and for medicine”, Maurizio Genuardi, director of the complex operational unit of Medical Genetics at the Gemelli Polyclinic in Rome and professor of Medical Genetics at the Catholic University, tells Adnkronos. As regards in particular the second aspect, “exploiting the mechanism of action of microRNAs Drugs have been produced that are used to treat some genetic diseasessuch as spinal muscular atrophy (Sma), and it has been possible to identify the basis of some genetic diseases which are due precisely to microRNA defects, such as the Dicer1 tumor predisposition syndrome. There are also further prospects for other applications, in particular for the early diagnosis of tumors and for other therapies also in the oncology field“.
On a scientific level, “the discovery has shed light on the functioning of DNA – highlights the expert – it has allowed us to discover a mechanism for regulating the function of genes. Until the discovery of microRNA, in fact, we knew the RNA that is produced from DNA and which in turn serves as a ‘mold’ for the synthesis of proteins. The merit of these scientists was to identify very small RNA molecules, which are always produced by DNA, but which are not used for synthesis. of proteins, but rather serve to modulate the activity of those parts of DNA that encode proteins”.
MicroRNAs have been known for some time now, clinical applications have emerged in recent years, but studies have been ongoing for a long time. It will still take time for other applications, but it is clear that they have opened a new path of studies in biology and medicine”.
For Genuardi, this award is “very important” as it recognizes the value of basic research. “It is important that this type of research receives adequate funding, because without it truly innovative ways of treatment or early diagnosis cannot be found”, he underlines. Ambros and Ruvkun’s discovery came from basic research, from a small worm, which helped them decipher human complexity. “Often – concludes Genuardi – fundamental research has been conducted starting from small organisms, such as the fruit fly, from these small vermicelli, rather than bacteria or yeasts”.
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