Long before mRNA vaccines became household terminology during the COVID-19 pandemic, researchers at the University of Rochester were quietly studying a molecule that much of the scientific world viewed as little more than DNA’s sidekick.
Today, those decades of work are helping shape some of medicine’s most promising frontiers, from cancer treatments and gene therapies to experimental approaches targeting rare genetic disorders. As RNA-based medicine rapidly expands, University of Rochester Medical Center scientists are finding themselves at the center of a scientific revolution they helped build.
Rochester’s scientists were studying RNA before it was popular
The recent explosion of interest in RNA research has been fueled by the success of COVID-19 vaccines, which relied on messenger RNA, or mRNA, technology to train the body’s immune system to recognize and fight the virus.
But the groundwork was laid decades earlier.
Lynne Maquat, founding director of the University of Rochester’s Center for RNA Biology, has studied RNA since 1972. She recalls a time when RNA research occupied the margins of scientific conferences and received far less attention than DNA research.
That has changed dramatically. More than 400 RNA-based drugs are now in development, billions of dollars have flowed into RNA-focused biotechnology companies, and recent Nobel Prizes have recognized discoveries involving RNA biology.
Researchers at Rochester were among those making foundational discoveries during those quieter years.
Research from Rochester helped make mRNA vaccines possible
One of the university’s most significant contributions came through work conducted by David Mathews and his mentor, Douglas Turner.
Beginning in the early 1990s, the pair developed what became known as the Turner Rules, a set of principles that help scientists predict how RNA molecules fold and behave. The shape of RNA is critically important because stable RNA molecules survive longer in cells, allowing them to produce more proteins and generate stronger immune responses.
Those principles became an important tool for companies developing mRNA vaccines during the pandemic. Moderna collaborated with Mathews before and during the COVID-19 crisis, using insights from his work to improve vaccine design.
What began as basic scientific curiosity decades ago ultimately became part of the scientific foundation that helped produce vaccines credited with saving millions of lives worldwide.
Decades of RNA research are now reaching patients
The impact of RNA science is increasingly moving beyond the laboratory and into clinics.
University researchers are helping develop treatments for conditions including myotonic dystrophy, a genetic disorder that causes progressive muscle weakness and can affect a person’s ability to walk, swallow and breathe.
Neurologists Charles Thornton and Johanna Hamel are involved in multiple clinical trials testing therapies that target toxic RNA molecules believed to drive the disease. Scientists say those studies represent the closest researchers have come to treatments capable of significantly changing the course of the illness.
Researchers are also investigating RNA-based approaches for rare genetic disorders, neurodevelopmental conditions and various forms of cancer.
Eric Wagner, a professor of biochemistry and biophysics, said advances in understanding RNA have transformed what is possible.
A decade ago, many of these diseases were viewed primarily as scientific puzzles. Today, researchers are discussing clinical trials and potential treatments.
A discovery from Rochester launched an entire field of study
Another major breakthrough emerged from Maquat’s work in the early 1980s.
While studying a rare blood disorder known as beta-thalassemia, she discovered that abnormal RNA metabolism—not DNA itself—was responsible for the disease. The finding became the first demonstration of a human disease caused by unstable messenger RNA.
Her research later led to the discovery of a cellular quality-control process called nonsense-mediated mRNA decay, or NMD. The mechanism helps cells identify and destroy faulty RNA molecules before they can produce harmful proteins.
Scientists now know that roughly one-third of genetic disorders involve mutations that can trigger this process.
The discovery launched an entirely new area of RNA biology and opened the door to potential therapies for conditions ranging from cystic fibrosis to Fragile X syndrome and other inherited diseases.
The future of medicine may be written in RNA
The University of Rochester is positioning itself to remain a major player in the next phase of RNA science.
In 2024, New York announced the creation of a new Center of Excellence in RNA Research and Therapeutics, a partnership between the University of Rochester and the University at Albany. The initiative will collaborate with biotechnology giants including Pfizer and Regeneron to develop new RNA-based treatments and train future researchers.
Plans are also underway for new research facilities designed to bring RNA scientists and cancer researchers together under one roof, accelerating efforts to develop therapies such as anti-tumor vaccines and precision genetic treatments.
Researchers say the field is only beginning to reveal its potential.
While much of the public’s attention focused on RNA during the pandemic, scientists at Rochester view that moment as a beginning rather than a culmination. After decades spent uncovering how RNA works inside cells, the next challenge is translating that knowledge into therapies that can change—and save—lives.
For the researchers who spent years studying a molecule few people had heard of, that future has finally arrived.
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