Two Massachusetts scientists receive Nobel Prize in Medicine for discovery of microRNA

Two Massachusetts scientists have been recognized with the Nobel Prize in Physiology or Medicine for their role in the discovery of microRNA — key to the understanding of gene regulation and potential treatments of heart disease, cancer, neurodegenerative diseases and more.

Researchers Victor Ambros, a University of Massachusetts Medical School professor of natural science, and Gary Ruvkun, a Massachusetts General Hospital and Harvard Medical School investigator and professor of genetics, received the Nobel Prize on Monday.

“Gene regulation by microRNA, first revealed by Ambros and Ruvkun, has been at work for hundreds of millions of years,” the Nobel Committee for Physiology or Medicine stated in a release. “This mechanism has enabled the evolution of increasingly complex organisms.”

The committee stated the scientists’ work “revealed an entirely new dimension to gene regulation” that are “proving to be fundamentally important for how organisms develop and function.”

In a press conference at MGH on Monday, Ruvkun called the study of recombinant DNA starting in the 70s a “revolution” and said as a young student and researcher he “just wanted to be part of that.”

In the late 1980s, Ambros and Ruvkun worked as postdoctoral fellows in the laboratory of Robert Horvitz, who was awarded the Nobel Prize in 2002. There they studied the 1 mm long roundworm, C. elegans, narrowing in on a mutation and gene function in the animals.

Ambros and Ruvkun continued the research after the fellowship at respectively at their Harvard University lab and Massachusetts General Hospital and Harvard Medical School lab. The pair compared findings, discovering the existence of microRNA in the worms, and published in 1993 in two articles in the journal Cell.

The discovery was met with “deafening silence from the scientific community,” the Nobel committee wrote, until 2000 when Ruvkun published new findings on microRNA in another gene, demonstrating their presence across the animal kingdom.

In the past two decades, “research into the potential of microRNAs for the diagnosis, prognosis and treatment of disease has expanded from the two original papers published by Ruvkun and Ambros in 1993 to 176,000 papers today,” MGH said in a statement.

The “unexpectedly short” microRNA, Ambros said, help regulate how genes are controlled in cells. The microRNAs “block gene expression by binding to regulatory segments in their target messenger RNAs,” MGH said.

Current research has shown human and most other plant and animal genomes contain “more than 1,000 microRNAs, which control many protein-coding messenger RNAs and may be involved in a broad range of normal- and disease-related activities,” the hospital said.

Researchers are currently conducting clinical trials involving microRNA for medical conditions including heart disease, cancer and neurodegenerative diseases.

Ambros said he was “surprised and delighted” to hear about the Nobel Prize at a press conference in the UMass Chan Medical School in Worcester on Monday and emphasized that studies of laboratory organisms of this kind are “critical and key and fundamental to advancing understanding of biology.”

“I think the unexpectedness of biology is probably the most important principle, perhaps, for people to appreciate,” said Ambros. … “At any given moment, it feels like we know most of what we need to know — that is actually an illusion that we have to consciously disabuse ourselves of and leave ourselves open for the surprises.”

The 2023 Nobel Prize in Physiology or Medicine was awarded to two researchers, Katalin Karikó and Drew Weissman, who helped develop mRNA vaccines to combat the COVID-19 pandemic.

Nobel prize announcements will continue with the physics prize on Tuesday, chemistry on Wednesday, literature on Thursday, Peace Prize on Friday and the Memorial Prize in Economic Sciences on Oct. 14.