From E=mc² to the division of the atom via the invention of the transistor, the first half of the 20th century was dominated by advances in physics.
Then, in the early 1950s, biology began to push physics out of the scientific spotlight – and when I say “biology,” I really mean DNA. The momentous discovery of the DNA double helix in 1953 more or less marked the beginning of a new era of science that culminated in the Human Genome Project, completed in 2003, which decoded all of our DNA into one biological model of humanity.
DNA has received immense attention. And while the double helix was certainly revolutionary in its time, the current generation of scientific history will be defined by a different (and, until recently, lesser known) molecule – one that I suspect will play a role yet more important in the advancement of our understanding. of human life: RNA.
You may remember learning about RNA (ribonucleic acid) in your high school biology class as the messenger that carries information stored in DNA to direct the formation of proteins. Such messenger RNAs, or mRNAs for short, have recently entered the mainstream debate thanks to the role they played in Covid-19 vaccines. But RNA is much more than a messenger, as critical as that function may be.
Other types of RNA, called “non-coding” RNAs, constitute a small biological powerhouse that can help treat and cure deadly diseases, unlock the potential of the human genome, and solve one of the most enduring mysteries of science: explaining the origins of all life. on our planet.
Despite being the keystone of every living thing on Earth, RNA has been misunderstood and underestimated for decades – often seen as nothing more than a simple biochemical singer, working in obscurity in the shadow of the diva, the DNA. I know this first hand: I worked in obscurity in his name.
In the early 1980s, when I was much younger and most of the promise of RNA was still unimagined, I opened my laboratory at the University of Colorado, Boulder. After two years of false leads and frustration, my research group discovered that the RNA we were studying had catalytic power. This means that RNA could cut and rejoin biochemical bonds on its own – the kind of activity that was thought to be the sole purview of protein enzymes. This gave us a tantalizing glimpse into our deepest origins: if RNA could both contain information and orchestrate the assembly of molecules, it was very likely that the first living things to emerge from the primordial ooze were organisms based on RNA.
This breakthrough in my laboratory – along with independent observations of RNA catalysis by Sidney Altman at Yale – was recognized with a Nobel Prize in 1989. The attention generated by this prize helped lead to an efflorescence of research which have continued to expand our idea of what RNA is. could do.
In recent years, our understanding of RNA has begun to advance even more rapidly. Since 2000, RNA-related breakthroughs have led to 11 Nobel Prizes. During the same period, the number of scientific journal articles and patents generated each year by RNA research has quadrupled. There are more than 400 RNA-based drugs in development, in addition to those already in use. And in 2022 alone, more than $1 billion in private equity funds have been invested in biotech startups to explore the frontiers of RNA research.
What drives the RNA era is the dazzling versatility of this molecule. Yes, RNA can store genetic information, just like DNA. As an example, many viruses (from influenza to Ebola to SARS-CoV-2) that plague us don’t care about DNA at all; their genes are made of RNA, which suits them just fine. But storing information is only the first chapter of the NRA playbook.
Unlike DNA, RNA plays many active roles in living cells. It acts like an enzyme, splicing and cutting other RNA molecules or assembling proteins – the stuff from which all life is built – from amino acid building blocks. It keeps stem cells active and prevents aging by building DNA at the ends of our chromosomes.
Discoveries about RNA have led to new therapies, such as the use of antisense RNA to help treat children with a devastating disease, spinal muscular atrophy. mRNA vaccines, which saved millions of lives during the Covid pandemic, are being reformulated to tackle other diseases, including some cancers. RNA research could also help us rewrite the future; the genetic scissors that give CRISPR its breathtaking power to modify genes are guided to their sites of action by RNA.
Although most scientists now agree on the brilliant promise of RNA, we are only just beginning to unlock its potential. Consider, for example, that about 75 percent of the human genome is made up of dark matter that is copied into RNAs of unknown function. Although some researchers have dismissed this dark matter as junk or noise, I expect it to be the source of even more exciting advances.
We don’t yet know how many of these possibilities will turn out to be true. But if the last 40 years of research have taught me anything, it’s to never underestimate this little molecule. The RNA era is just beginning.
Thomas Cech is a biochemist at the University of Colorado at Boulder; recipient of the Nobel Prize in Chemistry in 1989 for his work on RNA; and the author of “The Catalyst: RNA and the Quest to Unlock Life’s Deepest Secrets,” from which this essay is adapted.
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