January 17-18, 2023
The Herbert Wertheim UF Scripps Institute for Biomedical Innovation & Technology
Rodney B. Fink Auditorium
120 Scripps Way, Jupiter, Florida 33458
RNA: From Biology to Drug Discovery
See RNA Symposium Highlights and Photos
It was the first time many of the scholars had gathered since the pandemic began, and so there were many advances to share. Read an account of the conference and see a photo gallery.
Until recently, most researchers deemed RNA to be undruggable as a disease target. No more. The recent success of mRNA vaccines against COVID-19 show that RNA does not just cause disease. It can be both prevention and cure, as well.
Scientists and students are invited to visit The Herbert Wertheim UF Scripps Institute for Biomedical Innovation & Technology on Tuesday, Jan. 17 and Wednesday, Jan. 18 for a free symposium, “RNA: From Biology to Drug Discovery.” Hear from scientific luminaries who have revealed intriguing facets of the biology and structure of noncoding RNA, and meet some of the researchers who have challenged conventional wisdom to devise compounds that target, bind, edit and degrade disease-associated RNAs. Join a poster session to learn about new, emerging research in the field.
Speakers include experts in computation and biophysics; those uncovering intricacies of ribosomes, and those working toward treatment of diseases including myotonic dystrophy, ALS, Parkinson’s, cancer, Rett syndrome and viral diseases including COVID-19 and HIV. The universe of human proteins consists of about 20,000 varieties, while the universe of human RNAs is closer to 200,000, potentially offering myriad new opportunities to address genetic diseases with few or no options.
HHMI Investigator, Stephen W. Kuffler Chair in Biology; Department of Neurobiology; UC San Diego
Susan Ackerman, Ph.D.
Dr. Ackerman’s pioneering work using mouse genetics has identified novel genes, pathways, and networks involved in neurodevelopment and the maintenance of neuronal homeostasis and age-related death of neurons in the central nervous system.
Professor of Neurology, Biochemistry and Molecular Biology; Department of Neurology, University of Miami Miller School of Medicine
Antonio Barrientos, Ph.D.
Antonio Barrientos, Ph.D., is a professor of Neurology, Biochemistry and Molecular Biology with the Department of Neurology at the University of Miami Miller School of Medicine. He is also a member of the Biochemistry, Neuroscience and Pharmacology graduate programs, with secondary appointment to Biochemistry and Molecular Biology at the University of Miami.
Assistant professor, department of molecular medicine; The Herbert Wertheim UF Scripps Institute for Biomedical Innovation & Technology
James M. Burke, Ph.D.
James M. Burke, Ph.D., is an assistant professor of molecular medicine at The Herbert Wertheim UF Scripps Institute for Biomedical Innovation & Technology. Burke probes changes to RNA during the antiviral response to pathogenic viruses.
Virginia and D.K. Ludwig Professor of Cancer Research; Professor of Genetics Dermatology; Stanford University
Howard Chang, M.D., Ph.D.
Howard Y. Chang M.D., Ph.D., is director of the Center for Personal Dynamic Regulomes and the Virginia and D.K. Ludwig Professor of Cancer Research at Stanford University. He is a Howard Hughes Medical Institute investigator; he is also professor of Dermatology and of Genetics at Stanford University School of Medicine.
Max Planck Society President-Elect
Patrick Cramer, Ph.D.
Patrick Cramer, Ph.D., is president-elect of Germany's Max Planck Society. He currently directs the Max Planck Institute for Multidisciplinary Sciences. Professor Cramer studies how the genome is transcribed and regulated in health and disease, employing structural biology, bioinformatics and functional genomics methods. Dr. Cramer is a member of the German National Academy of Sciences Leopoldina, the European Molecular Biology Organization (EMBO) and in 2020 he was named to the U.S. National Academy of Sciences.
Professor and Chair, Department of Chemistry; The Herbert Wertheim UF Scripps Institute for Biomedical Innovation & Technology
Matthew D. Disney, Ph.D.
Matthew D. Disney, Ph.D., is professor and chair of the Department of Chemistry at The Herbert Wertheim UF Scripps Institute for Biomedical Innovation & Technology. The Disney group develops rational approaches to design selective therapeutics from only genome sequence.
Assistant Professor; Department of Molecular Genetics & Microbiology; University of Florida
Kotaro Fujii, Ph.D.
Kotaro Fujii, Ph.D., is an assistant professor of Molecular Genetics and Microbiology, and member of the Center for NeuroGenetics at the University of Florida College of Medicine.
Bloomberg Distinguished Professor of Molecular Biology; Johns Hopkins School of Medicine; Department of Molecular Biology and Genetics
Rachel Green, Ph.D.
An eminent microbiologist and geneticist, Rachel Green, Ph.D., is the Bloomberg Distinguished Professor of Molecular Biology and Genetics at Johns Hopkins University School of Medicine and a Howard Hughes Medical Institute investigator. She explores the role of ribosomes in translating the genetic information found in all cells.
Professor of Biochemistry and Chemical Engineering; Stanford University
Daniel Herschlag, Ph.D.
The overarching goal of Dr. Herschlag's research is to understand the fundamental behavior of RNA and proteins and, in turn, how these behaviors determine and impact biology and how it has evolved. His lab takes an interdisciplinary approach, spanning and integrating physics, chemistry and biology, and employing a wide range of techniques.
Professor of Integrative Structural and Computational Biology;The Herbert Wertheim UF Scripps Institute for Biomedical Innovation & Technology
Katrin Karbstein, Ph.D.
Katrin Karbstein, Ph.D., is professor of Integrative Structural and Computational Biology at The Herbert Wertheim UF Scripps Institute for Biomedical Innovation & Technology. Her research focuses on mechanisms of ribosome assembly, quality control and the molecular basis for ribosome-associated diseases (ribosomopathies).
Professor of Genetics and Molecular Biology; Massachusetts General Hospital and The Blavatnik Institute; Harvard Medical School
Jeannie Lee, M.D., Ph.D.
Jeannie T Lee is the Philip A. Sharp, Ph.D., Endowed Chair in Molecular Biology, Professor of Genetics (and Pathology) at Harvard Medical School, and vice chair of the Department of Molecular Biology at Massachusetts General Hospital. Lee specializes in the study of epigenetic regulation by long noncoding RNAs and uses X-chromosome inactivation as a model system.
Professor, Cech-Leinwand Endowed Chair of Biochemistry; University of Colorado Boulder
Roy Parker, Ph.D.
Roy Parker, Ph.D., is an investigator with the Howard Hughes Medical Institute; executive director of the BioFrontiers Institute; Cech-Leinwand Endowed Chair of Biochemistry and Distinguished Professor at the University of Colorado Boulder. He has a joint appointment with the departments of Molecular, Cellular and Developmental Biology.
Director, Center for RNA Biology, and Associate Professor, Department of Oncology, Johns Hopkins University School of Medicine
Ranjan J. Perera, Ph.D.
Dr. Perera is director of the Center for RNA Biology at Johns Hopkins All Children's Hospital, a senior scientist in the Cancer & Blood Disorders Institute and an associate professor of oncology in the Johns Hopkins University School of Medicine. He also has a secondary affiliation with the Johns Hopkins All Children’s Institute for Fundamental Biomedical Research.
Associate professor of immunology and microbiology;The Herbert Wertheim UF Scripps Institute for Biomedical Innovation & Technology
Matthew Pipkin, Ph.D.
The overall focus of the Pipkin lab is to elucidate how chromatin structure and transcription controls the gene expression programs that establish and maintain the differentiated states of T cells.
Sterling Professor of Molecular, Cellular and Developmental Biology and Chemistry; Yale School of Medicine
Anna Marie Pyle, Ph.D.
Anna Marie Pyle, Ph.D., is the Yale Sterling Professor in Molecular, Cellular and Developmental Biology and Chemistry, and she is a Howard Hughes Medical Institute investigator.
Director of the Center for NeuroGenetics, Professor, Molecular Genetics and Microbiology, University of Florida
Laura Ranum, Ph.D.
Dr. Ranum began her research career in human molecular genetics in 1989 at the University of Minnesota and is currently the founding director of the Center for NeuroGenetics and professor of Molecular Genetics and Microbiology at the University of Florida.
Center for Neurogenetics at the university of florida
Maurice S. Swanson, Ph.D.
Maurice S. Swanson, Ph.D., is professor and associate program director of Molecular Genetics and Microbiology at the University of Florida.
Professor and Associate Dean for Therapeutic Innovation; University of Miami Health System; Sylvester Comprehensive Cancer Center
Claes Wahlestedt, M.D., Ph.D.
Dr. Wahlestedt's laboratory pursues drug discovery and evaluation of combination therapies for various cancers. His team has been successful in identifying and bringing forward toward testing in humans several novel inhibitors of epigenetic and other drug targets. They are also conducting personalized ex vivo drug screening, with the cancer patient's own cells, to aid optimized treatment.
Associate Professor; Department of Molecular Genetics and Microbiology; University of Florida
Eric Wang, Ph.D.
Eric Wang, Ph.D., received his doctorate from Harvard-MIT Division of Health Sciences and Technology, working with mentors Christopher Burge and David Housman. There, he trained in computational biology, as well as learned about diseases such as muscular dystrophy, Huntington’s disease, and ALS.
Travel and Accommodations Information
Nearest airport: Palm Beach International Airport
- Directions to The Herbert Wertheim UF Scripps Institute for Biomedical Innovation & Technology
- Palm Beach County, Florida visitor information
- Courtyard by Marriott Palm Beach Jupiter
- Homewood Suites by Hilton Palm Beach Gardens
- Hampton Inn Jupiter/Juno Beach
Tuesday, January 17 | The Herbert Wertheim UF Scripps Institute for Biomedical Innovation & Technology | Rodney B. Fink Auditorium
|8:00 – 9:00 a.m.||Registration check-in and continental breakfast|
|9:00 – 9:10 a.m.||Matthew Disney, Ph.D.||Opening Remarks|
|9:10 – 9:40 a.m.||Matthew Disney, Ph.D.||Translating RNA Sequence Into Lead Small Molecule Medicines|
|9:40 – 10:10 a.m||Howard Chang, M.D., Ph.D.||Targeted RNA Delivery with Genomics|
|10:10 – 10:30 a.m.||Break|
|10:30 – 11:00 a.m.||Dan Herschlag, Ph.D.||RNA Beyond the Genomic Age|
|11:00 – 11:20 a.m.||Ranjan Perera, Ph.D.||Therapeutic Potential of Long noncoding RNA lnc-HLX-2-7 in Group III Medulloblastomas in Children|
|11:20 – 11:50 a.m.||Anna Marie Pyle, Ph.D.||Discovering and targeting the highly structured RNA regulatory elements that are unique to pathogenic fungi: a promising new route to well-tolerated systemic antimicrobials|
|11:50 – 1:30 p.m.||Lunch and Poster Viewing|
|1:30 – 2:00 p.m.||Susan Ackerman, Ph.D.||Transfer RNAs as Modifiers of Neurological Phenotypes|
|2:00 2:30 p.m.||Eric Wang, Ph.D.||Repeat Expansions Instigate Supply Chain Issues in the Nucleus and Cytoplasm|
|2:30 – 3:00 p.m.||Maurice Swanson, Ph.D.||Tandem Repeats in RNA Processing and Disease|
|3:00 – 3:30 p.m.||Break|
|3:30 – 4:00 p.m.||Claes Wahlestedt, M.D., Ph.D.||RNA targeted gene upregulation and therapeutic combination strategies|
|4:00- 4:30 p.m.||Antoni Barrientos, Ph.D.||The Human Mitochondrial mRNA Folding Landscape|
|4:30 – 4:50 p.m.||Allison Swain, Ph.D.||Mechanisms of the m64 RNA Modification in Meditating Cellular Switches, from Breast Cancer to Pathogenic Yeast|
|5:00 – 6:30 p.m.||Reception and Poster Viewing – The UF Scripps Café|
Wednesday, January 18 | The Herbert Wertheim UF Scripps Institute for Biomedical Innovation & Technology | Rodney B. Fink Auditorium
|8:00 – 9:00 a.m.||Registration check-in and continental breakfast|
|9:00 – 9:30 a.m.||Patrick Cramer, Ph.D.||Mechanisms of Chromatin Transcription|
|9:30 – 10.00 a.m.||James Burke, Ph.D.||Cellular RNA decay During Pathogenic Viral Infections|
|10:00 – 10:30 a.m.||Break|
|10:30 – 11:00 a.m.||Roy Parker, Ph.D.||RNP Granules in Health and Disease|
|11:00 – 11:20 a.m.||Michael Cortázar Osorio, Ph.D.||Nonsense-induced transcriptional upregulation: a model for future RNA therapies|
|11:20 – 11:50 a.m.||Matthew Pipkin, Ph.D.||RNA expression dynamics that specify protective T cell mediated immunity|
|11:50 -1:30 p.m.||Lunch and Poster Viewing|
|1:30 – 2:00 p.m.||Jeannie Lee, M.D., Ph.D.||Epigenetic Regulation by RNA G-quadruplexes|
|2:00 2:30 p.m.||Katrin Karbstein, Ph.D.||Quality Control of Ribosomes During and After Assembly|
|2:30 – 3:00 p.m.||Rachel Green, Ph.D.||The Colliding Ribosome as a Hub for Translational Regulation|
|3:00 – 3:30 p.m.||Break|
|3:30 pm – 4:00 p.m.||Laura Ranum, Ph.D.||Repeat associated non-AUG translation in neurodegenerative disease: mechanistic insights and therapeutic strategies|
|4:00- 4:20 p.m.||Alexey Petrov, Ph.D.||Sordarin Bound eEF2 Unlocks Spontaneous Forward and Reverse Translocation|
|4:20 – 4:50 p.m.||Kotaro Fujii, Ph.D.||Translation fidelity regulation by ribosome|
|4:50 – 5:00 p.m.||Matthew Disney, Ph.D.||Closing Remarks|
|5:00 – 6:30 p.m.||Reception – The UF Scripps Café|
Posters will be displayed near the café area and viewable during coffee breaks and lunch.
Matthew Disney Ph.D.
Chair, Department Of Chemistry
Katrin Karbstein Ph.D.
Maurice S Swanson
Professor And Associate Program Director
The organizers would like to thank UF Health, the academic health center of the University of Florida, for sponsoring this symposium, our second since 2019. Since our inaugural conference, the Scripps Florida campus has joined the University of Florida, a partnership that has brought new strengths and resources to both institutions. Read more here.
For Sponsorship questions, please contact Tracy Kerwin at firstname.lastname@example.org
We also gratefully recognize supporting sponsorships from: