JUPITER, Florida – More than 175 people attended “RNA: From Biology to Drug Discovery” at The Herbert Wertheim UF Scripps Institute for Biomedical Innovation & Technology on January 17 and 18, 2023. It was the first major scientific conference at the institute since the pandemic began, and so researchers relished the opportunity to share recent work and reconnect.
The conference attracted 18 impressive outside speakers, including multiple Howard Hughes Medical Institute investigators, members of the U.S. National Academy of Sciences and the Institute of Medicine. Incoming Max Planck President-Elect Patrick Cramer, Ph.D., shared his structural studies of the machinery underlying DNA transcription, featuring riveting imaging of transcription complexes in motion.
Faculty who conduct both basic and clinical studies involving RNA shared their discoveries in many areas. Until recently, RNA was deemed an unsuitable drug target by many in the scientific community. The speakers at the symposium showed how much the ground has shifted, as they described diverse projects aimed at fungal, viral, cancer and neurological disease targets, including epilepsy, Parkinson’s and ALS.
“The fact that 80% of the human genome encodes RNA while 1% encodes protein suggests that there is much to study about this biomolecule to leverage its huge potential to treat disease,” said symposium co-host Matthew Disney, Ph.D., who chairs the chemistry department at The Wertheim UF Scripps Institute.
Laura Ranum, Ph.D., the director of the Center for NeuroGenetics at the University of Florida, described her group’s research leading to a clinical trial of the drug metformin for treatment of C9orf72 ALS and frontotemporal dementia. The work may also have implications for Alzheimer’s disease.
Howard Chang, M.D., Ph.D., of Stanford University and HHMI, described how his group is harnessing AI and computational strategies to studies of long noncoding RNAs in a quest for ways to boost production of immune cells that can bind pathogens and cancer targets.
Maurice Swanson, Ph.D., associate program director at UF’s Center for NeuroGenetics and a co-host of the symposium, discussed his research on unstable genetic repeats in DNA and their role in diseases such as myotonic dystrophy type 1 and Huntington’s.
It was clear that advances in basic scientific understanding have been key to the recent rapid move toward clinical therapies. Many speakers shared new insights about DNA transcription and protein assembly via ribosomes. Cellular stress impacts and quality control mechanisms were a key focus.
Professor Katrin Karbstein, Ph.D., of The Wertheim UF Scripps Institute, a co-host of the conference, and Rachel Green, Ph.D., of the Johns Hopkins University School of Medicine and HHMI, discussed how cellular stress can lead to ribosomes collisions and protein assembly errors, a likely contributor to cell death and disease.
“Correct number and correct composition or ribosomes is a key to cell health,” Karbstein concluded.
Anna Marie Pyle, a professor at Yale University and HHMI and a past president of the RNA Society who is exploring development of antimicrobials that act on the organisms’ unique RNA, called the meeting a historic gathering of RNA leaders. She praised Disney for his pioneering work showing the druggability of RNA.
Organizers offered their special thanks to event sponsors UF Health, Alphazyme and Sarepta Therapeutics. They said plans are in the works for a repeat of the symposium in 2025.
Three Symposium Poster Presenters Take Home Awards
Conferences hold poster sessions to foster science communications and presentation skills among scientific trainees. At “RNA: From Biology to Drug Discovery,” trainees had the opportunity to showcase their work to some of the field’s leading scientists. Three trainees took home awards.
Ryan Hildebrandt, Ph.D., a postdoctoral researcher with associate professor Eric T. Wang, Ph.D., at the University of Florida in Gainesville, took home a poster award for his studies of how RNA moves within cells by riding other molecules, kinesins, enabling localized protein construction in cells.
Ebba Blomqvist, an FAU graduate and research technician with professor Katrin Karbstein, Ph.D., of The Wertheim UF Scripps Institute, described discovery of how an assembly factor helps make ribosomes, the cellular organelles that build proteins.
Jessica Bush, a doctoral student working in the Disney lab, won a poster award for her work designing a small, drug-like molecule to degrade an RNA that causes ALS and frontotemporal dementia.
Institute benefactor Dr. Herbert Wertheim presented their certificates, praising them for hard work and dedication to expanding knowledge, as they work for the betterment of humanity.