Rna: an acronym that until two years ago was mysterious for many, and which today instead we all look at with respect and gratitude given that the science of RNA is at the basis of vaccines against Covid. And that RNA, ribonucleic acid, is today the protagonist of a new world of medicine, a new way to develop therapies for many diseases. There is one of the people who started this revolution Craig Cameron Mellowho in 2006 won the Nobel Prize and on 20 October will be in Rome on the stage of the Health Festival, interviewed by Maurizio Molinarito tell us about the medicine to come.
In detail: Mello discovered gene silencing via the RNA interference mechanism, a technology that offers enormous prospects for creating a range of selective drugs against harmful genes. And to turn his research into cures, the volcanic scientist founded the biotech startup Atalanta Therapeutics, in honor of the Greek heroine. A name that, however, was enough for the fans of the Bergamo team to get to give him the Josip Ilicic t-shirt with number 72. In short, a Nobel Prize in black and blue.
But that Nobel Prize, Mello risked losing him, because of what, together with biology, is the passion that makes his heart beat: sailing. The day before receiving the call from Stockholm, in fact, the scientist had capsized with his beloved sailboat in Narragansett Bay and had been rescued by another boat that was nearby.
Surfing has always been liked by that little boy from Connecticut, born in 1959, scholastically lazy, who, however, barely reached the age of forty, in 1998, as professor of molecular medicine at the University of Massachusetts Medical School in Worcester (a chair that is his still today), to get your hands on an extraordinary discovery. He does so together with his rival Andrew Fire, a researcher at the Carnegie Institution of Washington, who is eager like him to find ways to intervene on genes to tackle incurable diseases. The Eureka find it together, when rivalry becomes collaboration.
The path of genetic manipulation was uphill and full of unsettling unexpected events: years earlier Richard Jorgensen, wanting to make the color of red petunias more intense, had inserted an extra copy of the gene that controls pigment formation into the cells of the flower; but instead of a brighter red, half of the second generation petunias were white. Because? Mello understood this thanks to a transparent millimeter-long worm, the C. elegans. He injected into the gametes of C. elegans various combinations of pieces of Rna with the code necessary to produce a protein crucial for movement. And he saw that by injecting a particular combination of Rna, he switched off that gene in the worm’s descendants, who therefore moved in jerks. Mello and Fire understood the mechanism: the two complementary strands, once injected into a cell, join together to form a double-stranded RNA, and it is this double molecule that is able to silence genes. It is a natural mechanism used by the most basic organisms, without an immune system, to defend themselves against viruses by deactivating the genes used by invaders to replicate. The discovery of Mello and Fire has allowed therapies that remedy diseases caused by the hyperactivity of a gene, “silencing” it. We have drugs – already on the market – that lower cholesterol or cure hepatic porphyrias, and promising still experimental treatments for Alzheimer’s. “Today when people get sick, whether from cancer or from genetic or developmental diseases, we can not only treat the disease, but also find out what is wrong with the genetic information that is not properly encoded by the cells,” he said. Mello. “And this gives us the opportunity to cure the patient by correcting the genetic information.”