The Nobel Prize in Medicine was awarded this year jointly to Victor Ambros and Gary Ruvkun for the discovery of microRNAs and their role in post-transcriptional gene regulation. This year’s Nobel Prize focuses on the discovery of a vital regulatory mechanism used in cells to control gene activity. Genetic information flows from DNA to messenger RNA (mRNA), via a process called transcription, and then to the cell’s protein-making machinery. There, mRNAs are translated so that proteins are produced according to genetic instructions stored in DNA. Since the mid-20th century, several of the most fundamental scientific discoveries have explained how these processes work. Our organs and tissues are made up of many different cell types, all with the same genetic information stored in their DNA. However, these different cells express unique sets of proteins. How is this possible? The answer lies in precisely regulating gene activity so that only the correct set of genes are active in each specific cell type. In the 1960s, it was shown that specialized proteins, known as transcription factors, can bind to specific regions of DNA and control the flow of genetic information by determining which mRNAs are produced. Since then, thousands of transcription factors have been identified, and it was long believed that the fundamental principles of gene regulation had been resolved.
However, in 1993, this year’s Nobel laureates published unexpected results describing a new level of gene regulation, which turned out to be highly significant and conserved throughout evolution. They discovered microRNAs, a new class of tiny RNA molecules that play a crucial role in gene regulation. Their groundbreaking discovery revealed an entirely new principle of gene regulation that has proven essential for multicellular organisms, including humans. It is now known that the human genome encodes over a thousand microRNAs.
Their surprising discovery revealed a completely new dimension of gene regulation. MicroRNAs are proving to be fundamentally important to how organisms develop and function. Gene regulation via microRNA, first revealed by Ambros and Ruvkun, has been in place for hundreds of millions of years. This mechanism has allowed the evolution of increasingly complex organisms. Abnormal regulation via microRNAs can contribute to cancer, and mutations in genes encoding microRNAs have been found in humans, causing conditions such as congenital hearing loss, ocular and skeletal disorders.
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