, director of the Ģ����ý Center for RNA Biology, is the winner of the  and the . Honored for her research on RNA mechanisms that contribute to a wide range of diseases, Maquat’s work has laid the foundation for the development of treatments targeting conditions that can’t always be corrected with conventional drugs, such as cystic fibrosis, cancers, and fragile X syndrome.

The J. Lowell Orbison Endowed Chair and Professor in the Department of Biochemistry and Biophysics at the URochester School of Medicine and Dentistry, Maquat has spent her career deciphering the many roles that  and is well known for her discovery of nonsense-mediated mRNA decay, or NMD. One of the major surveillance systems in the body, NMD protects against innate mistakes in gene expression by targeting and eliminating deleterious mRNAs that could lead to the production of incomplete and potentially toxic proteins that can cause disease.

Maquat shares the Dr. Paul Janssen Award with Alexander Varshavsky of the California Institute of Technology, and the Albany Prize with Howard Chang of Stanford University School of Medicine, and Adrian Krainer of Cold Spring Harbor Laboratory. Past winners of both awards have gone on to win the Nobel Prize.

“Lynne’s scientific career is nothing short of outstanding and her contributions have brought RNA biology to the leading edge of medicine,” says David Linehan, CEO of the Ģ����ý Medical Center and dean of the School of Medicine and Dentistry. “These back-to-back awards acknowledge her exceptional ability to discover, innovate, and push the limits of scientific understanding, and we’re extremely proud that her efforts are being acknowledged with these major honors.”

• Learn more about Maquat’s and

The Ģ����ý and University at Albany are partnering on a new Center of Excellence in RNA Research and Therapeutics focused on developing RNA-based therapies and training the next generation of New York’s biotechnology workforce.

Assemblymember Harry Bronson (D-Rochester) led the charge to secure funds in the 2024–25 New York State Enacted Budget to establish the new Center of Excellence, known as CERRT, which will initially receive $250,000.

Home to renowned scientists with decades of experience and millions of dollars in external funding for RNA-based research, the institutions will work together to promote economic growth through continued scientific discovery and partnership with industry.

RNA treatments hold enormous promise

The field of RNA science has catapulted to the forefront of health and medicine over the past decade as the work of leading scientists like and , the respective directors of the and , and others revealed the role that RNA plays in a multitude of diseases.

Myotonic dystrophy, cystic fibrosis, fragile X syndrome, and many other inherited disorders can be targeted with RNA-based treatments. The COVID-19 pandemic, meanwhile, highlighted the power of RNA to prevent disease as the development and deployment of mRNA vaccines saved untold lives and helped the US economy safely reopen.

 
“Our scientists have deep experience and a proven track record of important discoveries that are propelling the fast-growing field of RNA biology forward,” says Ģ����ý Vice President for Research . “Together with our partners in Albany, we’re poised to use our knowledge and expertise to build up this Center of Excellence, bringing new discoveries closer to patients and contributing to the state’s growth as a hub for health and technology research and development.”

“The COVID-19 pandemic focused the world’s attention on the importance of RNA science and the way strategic public investments and thoughtful industry partnerships can lead to new therapies that alleviate suffering and improve quality of life across a range of diseases,” says University at Albany President Havidán Rodríguez. “Thanks to groundbreaking research by scientists in Albany and Rochester who have been leading this work for decades and dogged advocacy by Assemblymembers Fahy, McDonald, and Bronson, UAlbany and Rochester will continue to advance cutting-edge RNA science that provides high-tech jobs for New Yorkers while helping people live longer, healthier lives.”

New state funding launches center

The CERRT joins 14 other centers based at universities across New York in the . Managed by ’s (ESD) Division of Science, Technology, and Innovation, the centers establish and advance collaborations between the academic research community and the business sector to develop and commercialize new products and technologies; promote critical private sector investment in emerging scientific fields in New York; and create and expand businesses and employment.

“Supporting an equitable and diverse array of research is critical to the success and longevity of biotechnology and pharmaceutical companies in New York, but also to the economic vitality of communities throughout New York,” says Assemblymember Bronson, who is chair of the Committee on Labor and represents the Ģ����ý Medical Center in the Assembly. “These centers attract worldwide talent to participate in and develop innovative, life-saving research. Rochester and Albany are already home to world-renowned medical researchers in the RNA field, and an RNA-focused Center of Excellence will give biotechnology and pharmaceutical companies—large and small—the competitive advantage they need to succeed by providing them access to leading researchers, cutting-edge technology, and a pipeline of top talent.”

“This new center is a win all around,” says , CEO of the Ģ����ý Medical Center and dean of the School of Medicine and Dentistry (SMD). “Patients and families will benefit from groundbreaking work carried out between researchers in Rochester and Albany; our trainees will gain invaluable experience working with experts in university labs and in industry; and New York will emerge as a leader in this vital field. We are grateful to Assemblymember Bronson for his efforts and the governor for her support.”

“This new Center of Excellence will strengthen critical ties between academic RNA research centers and biotechnology industry leaders and ensure New York’s biotech sector has the skilled workforce it needs to continue growing and innovating,” says Thenkurussi (Kesh) Kesavadas, the vice president for research and economic development at Albany. “Combined with UAlbany’s investments in AI supercomputing, the research and training conducted through CERRT holds enormous potential to develop RNA-based therapies for diseases impacting tens-of-thousands of New Yorkers.”

Building New York’s biotech workforce

CERRT will work with large New York biotech companies like Regeneron, Pfizer, and Curia to develop new therapeutics and to establish a pipeline of trained workers. According to Maquat, one of the first orders of business is to establish programs where SMD trainees engage in short-term “sabbaticals” at these companies, learning how industry operates and understanding the various roles scientists play in the private sector.

“A major goal of academic centers like Rochester and Albany is to provide our students and postdoctoral fellows with the best training possible, so they have the skills needed to pursue the career path of their choice,” says Maquat, the J. Lowell Orbison Distinguished Service Alumni Professor and a professor of biochemistry and biophysics, pediatrics, and oncology. “The Center of Excellence will ensure our trainees are competitive in the job market and help us recruit strong candidates to study and conduct research at our institutions in the future.”

Maquat will co-lead the CERRT at the URochester with , a professor of biochemistry and biophysics and a member of Rochester’s Center for RNA Biology, while Berglund with , a distinguished professor of biological sciences and a member of the University at Albany’s RNA Institute, will co-lead the CERRT at Albany.

“Training the next generation of RNA scientists is central to the mission of UAlbany’s RNA Institute, and we are excited to partner with our Ģ����ý colleagues to meet the workforce needs of New York industry and develop treatments caused by defective RNAs,” says Berglund, who is also the Keith Hynes Endowed Professor in STEM at the University at Albany.

Support for the Center of Excellence in RNA Research and Therapeutics

Partners and collaborators also expressed their support for the new center:

Jennifer Hawks Bland, NewYorkBIO CEO: “Supporting research in life sciences is critical to the success and longevity of all biotechnology and pharmaceutical companies across New York. CERRT will provide valuable research and workforce development collaborations throughout our industry, while also helping to attract, support, and retain talent in New York at a pivotal time for our state. This new Center of Excellence will further enhance future generations’ ability to research, develop, and commercialize life-saving technologies and therapeutics for patients in New York and beyond.”

Hope Knight, Empire State Development Commissioner and CEO: “Empire State Development is strategically focused on strengthening the innovation economy and NYSTAR’s Centers of Excellence are essential to our mission. This new partnership between UAlbany and the Ģ����ý will encourage collaboration between academia and the private sector and will provide an environment that will commercialize new products and technologies right here in New York State”

Emily Jones, myotonic dystrophy group facilitator in the Western New York Finger Lakes Region: “First and foremost, we want to thank Assemblymember Harry Bronson for listening to what it is like for our families to live day-to-day with myotonic dystrophy, a rare form of muscular dystrophy. He compassionately listened to caregivers and affected individuals share their daily challenges in the face of overwhelming fatigue, loss of the ability to work, and continuous pain. The addition of a NYS Center of Excellence for RNA-extended repeat diseases will fund world-class leaders at the URochester and University at Albany. Their research understanding will help pharmaceutical companies target the toxic RNA and the mechanisms that are the root cause of the disease. As a result, these companies will bring a therapeutic drug that will slow or stop disease progression of this brutal disease. Our families live with the hope of a cure that will come more quickly.”

Lois Schenk, myotonic dystrophy group facilitator in the Western New York Finger Lakes Region: “Treating and curing this rare disease is critical, certainly to families like mine and Emily’s, but also to the residents and government of New York State, as it is estimated that the economic burden of caring for individuals with this disease can cost the state upwards of $2 million per year.”

CRISPR regularly graces health and medicine headlines, with no sign of stopping. Within the sprawling field, Ģ����ý professor and researcher is creating a niche by zeroing in on CRISPR-Cas13. His goal? To better understand prokaryotic antiviral immunity and RNA-mediated gene regulation, and to inform the development of RNA detection tools that could improve clinical diagnostics. The assistant professor of biochemistry and biophysics recently published two in Nucleic Acids Research, and in a found that this same Cas13 protein partners with a unique membrane protein to stop viral infection.

O’Connell joined the University’s in 2017. Get to know him better as he shares his professional and personal path to Rochester.

WHAT’S UNEXPECTED ABOUT YOUR TWO NEWEST PAPERS?

��※��DzԲԱ����: We went in trying to understand allostery—how something that happens at one site in a protein (say, a drug binding) affects what happens at a different, distant site in the same protein. We identified a central communications hub and discovered that single amino acid substitutions (in what is a huge protein) altered the transmission of information across the protein. We were shocked that such a simple change made a difference and were able to use this strategy to create new variants of Cas13 that are more sequence-specific with respect to RNA recognition, and show these can improve our ability to discriminate between closely related SARS-CoV-2 strains using a CRISPR-based diagnostic test.

How’d you get into the CRISPR field?

��※��DzԲԱ����: I’m originally from Australia and got my PhD in Sydney, but wanted to do a postdoc in the US. I contacted a bunch of people, including Jennifer Doudna, to get interviews. I didn’t hear back, so I emailed Jennifer again and she asked me to visit her at Berkeley. This was back in 2012, and a week after our correspondence she published the Science study on CRISPR-Cas9 that won her the 2020 . I guess it was serendipitous that she replied to my email and I was able to join her lab; it was a really inspiring and invigorating time to be there.

Outside of your own, what areas of research do you find most interesting?

��※��DzԲԱ����: Many things! I loved learning about astrophysics and the universe as a kid and went to college with the hopes of becoming an astrophysicist. However, in my first year, I fell in love with protein structure and function and quickly decided that’s the path I wanted to take.

In terms of recent research, I’ve been fascinated with how we have greater than 1,000 different odorant receptor genes in our genome but each of our approximately 10 million olfactory neurons decides to randomly select and express just one gene from this gene family. How this regulation is thought to occur is fascinating and still a bit of a mystery, although scientists have made some .

What’s your favorite thing about Rochester?

��※��DzԲԱ����: How easy it is to live and work here, thanks to the quick commute and affordable housing. In terms of the University, I think our collaborative nature, collegiality, and support from the institution stand out as our biggest strengths. As do all of our undergraduate and graduate students—they are excellent.

When you aren’t in your office or lab, where can we find you?

��※��DzԲԱ����: My two-and-a-half year old is obsessed with ice hockey, so I’m usually taking him to skating lessons. I’m also a big music head; I love seeing local bands in the area, or traveling to shows in Buffalo or Syracuse. I have a pretty extensive record collection, so I spend time buying and selling. I like most kinds of music, but have a particular penchant for anything that is dark, mysterious, and noisy.

More from Mitch O’Connell

An appeared this weekend in the Democrat and Chronicle discussing the critical role that federal-funded research plays in improving people’s lives, driving economic growth, and making the nation a global leader in scientific innovation.

This progress and the transformative research being conducted here at the University and elsewhere are threatened by the deep cuts in research proposed under the President’s FY18 budget.

If you missed it, please also take the time to read the message from President Joel Seligman, Provost Rob Clark, and Medical Center CEO Mark Taubman on this topic.

March for Science
As many of you are aware, events are being planned across the country this weekend in support of research.  The is a non-partisan international movement, led by organizers around the globe to sound a call to support and safeguard the scientific community. Among many Ģ����ý people taking part in the D.C. march, physics professor Adam Frank will be participating in a teach-in with planetary scientists David Grinspoon and Ellen Stofan titled “Climate Change and the Cosmic Perspective.” Frank has written about the march on the NPR blog 13.7 cosmos & culture. You can read his posts and .

Local organizers are planning a Rochester-based march to be held concurrently with the national march on April 22. The culminates in a at the Hyatt Regency.  Members of the Ģ����ý community will also take part.

“The March for Science is an excellent opportunity for faculty, staff and students throughout the University to share their individual views on the tremendous impact that research has on the human condition globally,” says David Williams, dean for research in Arts, Sciences and Engineering at the University.

“I’ll be speaking at the Rochester Science Expo and participating in the local march, as an individual and as a scientist, because I want to communicate the importance of science to our society.  As scientists, I feel that we haven’t done enough to explain the value of what we do – from fundamental discoveries, to new treatments and cures (such as the HPV vaccine), to economic impact and job creation,” notes Stephen Dewhurst, vice dean for research at the School of Medicine and Dentistry. “We are 3,000 strong at the Medical Center alone, and we are working to improve life for everyone.  I see the march as an opportunity to celebrate science and scientists, to connect with the public at large, to express our appreciation for the support that scientific research has received in this country over the last half century, and to express the need to continue that support in the future.”

“I’m an introvert; activism and community outreach are way outside my comfort zone. But, when I learned about the march and expo, there was no question in my mind that I would be a part of it. My fellow trainees and I won’t have careers if we don’t work to keep science a priority in this country,” says Emma Grygotis, a fourth year graduate student in pharmacology and physiology at the School of Medicine and Dentistry, who is helping organize both events. “We want to encourage people in the community to join us. We all bring unique stories to this conversation, and the more voices we have, the stronger we’ll be.”

Pedro Benitez is a consumer, advocate for the Latino community and member of the UR HIV/AIDS Clinical Trials Unit Community Advisory Board. He’s participated in clinical research and is supporting the march because he wants to help physicians and scientists find a cure for HIV. “Without federal funding for research, how can we find a cure and help people live longer and better lives?” he asks.

Congress will take up the topic of funding for science and basic research in the coming months and there will likely be additional opportunities for people to advocate in support of these issues.

You can find more information about the President’s proposed budget, leadership advocacy in support of science, and the on the .