Q&A: Unpacking a Big Win for RNA Research

Seven bottles of the covid-19 vaccine are on a smooth white tabletop with blurred background.

Photo by Braňo / Unslpash.com

ALBANY, N.Y. (Oct. 10, 2023) — This year’s Nobel Prize in Medicine was awarded to scientists Katalin Karikó and Drew Weissman for laying the scientific foundation that enabled the development of the mRNA vaccine for COVID-19. The vaccine has saved millions of lives and served as a critical first step toward ending the coronavirus pandemic.

At the heart of the work: basic RNA research. The Nobel recognition is a testament to the importance of advancing RNA science and its potential to revolutionize the way we diagnose, treat and protect against a wide variety of diseases.

Andy Berglund, wearing a button-down shirt, is pictured standing in his lab in front of a shelf containing plastic bottles and glass test tubes. — Andy Berglund, professor and director of the RNA Institute.

Among those with a deep appreciation for this type of work is Andy Berglund, professor of biological sciences at UAlbany and director of the RNA Institute. Berglund’s research explores mechanisms that affect the activity of RNA binding proteins, which can lead to errors in RNA splicing that cause disease, and developing small molecules that target toxic RNAs to stop splicing errors.

We asked Berglund to explain the scientific discovery behind this year’s Nobel Prize in Medicine, what made the work revolutionary and the importance of advancing RNA science for applications in medicine and other fields.

What did Katalin Karikó and Drew Weissman discover, and how did that allow for the development of the mRNA vaccine for COVID-19?

The research underpinning the mRNA vaccine for COVID-19 has been in development for decades and remains an important area of ongoing research that could be used to protect against many other illnesses, including certain types of cancer. The remarkable efforts by Katalin Karikó and Drew Weissman being recognized by this year’s Nobel Prize demonstrate the necessity of basic science and the need to continue exploring the powerful biomedical potential of RNA technologies to fight disease, improve human health and save lives.

Central to Karikó and Weissman’s work is the relationship between messenger RNA (mRNA) — the genetic material that tells cells how to make proteins — and the body’s immune response. Karikó and Weissman revealed that it is possible for modified mRNA to be taken up by cells without provoking an immune response. In the case of the mRNA vaccine for COVID-19, this makes it possible to inject mRNA into the body, then cells read the instructions from this mRNA and make the spike protein from the SARS-CoV-2 virus that causes COVID-19. When your body encounters this spike protein on the surface of SARS-CoV-2 virus, it will recognize it and know how to fight it off, preventing severe illness.

What makes the research being honored by the Nobel Prize groundbreaking?

The groundbreaking aspect of the research done by Karikó and Weissman was to demonstrate that it is possible to incorporate naturally occurring RNA modifications found in human cells into mRNAs made in the lab, and that these modified mRNAs can be used to significantly reduce the immune response. It was this critical result that allowed for the development of the COVID-19 vaccines and likely many future medical breakthroughs.

The fundamental RNA research done by Karikó and Weissman allowed Pfizer, BioNTech and Moderna to rapidly generate mRNA vaccines that saved millions of lives. Without the RNA technology that Karikó and Weissman had worked for decades to develop, it would have taken much longer for vaccines to be produced, and the impact of COVID-19 on the world would have been even more significant.

This technology will be used to fight future viral outbreaks. Scientists at universities, biotech and pharmaceutical companies are developing mRNA and other RNA approaches that will be used to fight cancer, muscular dystrophies and many other human diseases. For cancer, one of the approaches is to use mRNAs to activate the immune system to kill cancer cells.

Why is RNA research important? What answers could it hold?

RNA modifications are an important area of research with implications for human development and disease. There are more than 150 RNA modifications found in humans, animals, bacteria and generally across all organisms.

An area of focus at UAlbany’s RNA Institute is to understand the function of RNA modifications and how these modifications could be harnessed not only for biomedical applications, but for future RNA technologies that could help solve challenges in other fields as well. Potential uses of RNA technologies include the development of drought and viral-resistant crops. RNA science is also being explored as a way to increase carbon uptake in plants and bacteria to help fight climate change. RNA science can have broad impacts beyond human disease.