Two US scientists, Victor Ambros and Gary Ruvkun, have won the 2024 Nobel prize in medicine for their discovery of microRNA—a new class of tiny ribonucleic acid (RNA) molecules that play a crucial role in gene regulation, unfolding how multicellular organisms live.
Their work explains how cells develop into different types—such as those in muscles and nerves—although all cells in an individual contain the same set of genes and instructions for growth. More specifically, they discovered the role microRNA played in this process. RNA is essential for most biological functions, setting a template for production of proteins, the building blocks of all animal life. When a single-strand messenger RNA is decoded and translated into making proteins, microRNA comes into play.
Ruvkun describes microRNAs as a “communication network among genes”, enabling our cells to generate complex structures and functions. Together, the two scientists have revealed a new principle of gene regulation that turns out to be essential for multicellular organisms. It is fundamental to how organisms develop and function. They found how a single microRNA can regulate the expression of many different genes, and conversely, how a single gene can be regulated by multiple microRNAs, thereby coordinating and fine-tuning entire networks of genes to contribute to cell formation, and in turn committing to form different organs, muscles and tissues in the body.
Modern medical professionals are increasingly familiar with genetic predisposition to diseases—the tendency of people developing particular diseases in the future based on their genetic makeup. Diabetes, heart disease, asthma, cancer and single-gene disorders are some of them. The Ambros-Ruvkun research reveals that cells and tissues cannot develop normally without microRNAs. The flipside: abnormal regulation by microRNA can contribute to cancers; mutations in genes coding for microRNAs can cause congenital hearing loss, eye and skeletal disorders; and mutations in a protein required for microRNA production can result in the DICER1 syndrome, a rare but severe syndrome linked to cancer. The medical science community considers this discovery a major breakthrough with far-reaching consequences including newer treatments—and, potentially, prevention—for a whole range of diseases through gene therapy. So the Nobel-winning find is a major shot in the arm for the medical fraternity in particular and humanity in general.