Member Directory for The Center for RNA Genomic Medicine

Jessica Childs-Disney

Jessica Childs-Disney

RES AST PROF
Department: SR-CHEM-SHEN LAB
Phone: (561) 228-2508
Mailing Address:
Publications:
Grants:
  • Oct 2025 ACTIVE
    Novel mRNA degraders to target hypoxic tumor microenvironment
    FL DEPT OF HLTH BANKHEAD-COLEY CANCER RE · Co-Project Director/Principal Investigator
  • Feb 2025 – Jan 2026
    ASD gene therapy: Large-scale evaluation of small-molecule targeting of RNA for ASD therapeutics
    ICAHN SCHOOL OF MEDICINE AT MOUNT SINAI · Principal Investigator
  • Jun 2024 – Dec 2025
    Developing Artificial Intelligence and Machine Learning Approaches to Identify and Optimize Small Molecule Medicines Targeting Oncogenic RNAs
    FL DEPT OF HLTH · Principal Investigator
  • Mar 2024 ACTIVE
    Design of small molecules that degrade the mRNA encoding a-synuclein
    SPARK NS · Co-Project Director/Principal Investigator
  • Apr 2022 – Nov 2023
    Advance Therapeutic Targets & Biomarkers for ALS FTD
    TARGET ALS FOUNDATION · Principal Investigator
  • Apr 2022 – Feb 2024
    Targeting RNAs Encoding Proteins Associated with Autism Spectrum Disorders
    ICAHN SCHOOL OF MEDICINE AT MOUNT SINAI · Principal Investigator
Matthew Disney

Matthew Disney Ph.D.

Institute Professor & Chair, Department of Chemistry
Department: SR-CHEM-DISNEY LAB
Phone: (561) 228-2203
Mailing Address:
Physical Address:
Accomplishments:
  • Recipient
    2021 · The ACS 2022 Nobel Laureate Signature Award for Graduate Education in Chemistry
  • Awarded
    2019 · The Raymond and Beverly Sackler International Prize in Chemistry
  • The Barry Cohen Prize
    2018 · Medicinal Chemistry Section of the Israel Chemical Society and Teva Pharmaceutical Industries.
  • Awarded
    2018 · BioFlorida's Weaver H. Gaines Entrepreneur of the Year
  • Outstanding Mentor Award
    2017 · Scripps Florida
  • Tetrahedron Young Investigator Award in Bioorganic and Medicinal Chemistry
    2016 ·
  • Blavatnik Young Scientists Award Finalist
    2015-2017 ·
  • NIH Director’s Pioneer Award
    2015 · National Institutes of Health
  • David W. Robertson Award in Medicinal Chemistry
    2014 · American Chemical Society’s Division of Medicinal Chemistry
  • Excellence Award in the field of Research in Science and Technology
    2013 · India-US Chamber of Commerce, Inc.
  • Eli Lilly Award in Biological Chemistry
    2013 · American Chemical Society
  • David Gin Award in Carbohydrate Chemistry
    2012 · American Chemical Society
  • Dreyfus Teacher-Scholar Award
    2010-2015 ·
  • Excellent Scholar, Young Investigator Award
    2010 · University at Buffalo
  • Research Corporation Cottrell Scholar Award
    2008-2010 ·
  • NYSTAR JD Watson Young Investigator Award
    2007-2009 ·
  • Camille and Henry Dreyfus New Faculty Award
    2005-2010 ·
  • Second Year Roche Foundation Postdoctoral Fellowship
    2004-2005 · Swiss Federal Institute of Technology
  • Roche Foundation Postdoctoral Fellowship
    2003-2004 · Swiss Federal Institute of Technology
  • Arnold Weissberger Memorial Fellow
    2001-2002 · University of Rochester
  • Elon Huntington Hooker Memorial Fellow
    2000-2001 · University of Rochester
  • Sherman-Clark Memorial Fellow
    1997-1999 · University of Rochester
  • Eric A. Batista Award
    1997 · University of Maryland at College Park
  • Howard Hughes Medical Institute Undergraduate Research Fellow
    1995-1997 · University of Maryland at College Park
Research Summary:

The Disney group develops rational approaches to design selective therapeutics from only genome sequence. One of the major advantages that genome sequencing efforts potentially provides is advancing patient-specific therapies, yet such developments have been only sparsely reported. We have developed general approach to provide lead Targeted Therapeutics and Precise Medicines that target RNAs that cause disease broadly and include rare neuromuscular (muscular dystrophy), neurodegenerative (Alzheimer’s, ALS), infectious diseases as well as difficult-to-treat cancers (breast, pancreatic, prostate, and others), and infectious diseases that can emerge through seasonal exposures. Designed compounds have demonstrated activity in human derived cellular disease models as well as pre-clinical animal models of disease. We train the next-generation of scientists to ensure our work has an exponential impact in studying disease biology and leveraging it into making Precision Medicines.

To achieve these goals, we developed a proprietary platform dubbed Inforna over the past 13 years. It merges chemoinformatics and RNA structure to identify lead compounds that target an RNA of interest; that is, Inforna houses a database of RNA three dimensional motifs that bind small molecule medicines, identified via an experimental library-versus-library screen. The bioinformatics pipeline rapidly and accurately identifies disease-associated RNA sequences that adopt targetable three-dimensional folds by comparison to the database. This pipeline has been validated in various peer-review publications that demonstrated that the platform can be used to target RNAs that cause neuromuscular, neurodegenerative, and infectious diseases as well as difficult-to-treat cancers in pre-clinical animal models. Additionally, lead small molecule medicines can also be rapidly developed into compounds that recruit cellular nucleases to selectively destroy the RNAs that cause these diseases in a catalytic and substoichiometric manner (e.g. one molecule of the small molecule cleaves more than one molecule of the RNA target) coined RIBOTACs. Two of the major perceived concerns in the area of RNA-targeted small molecules are selectivity and potency. We have broadly demonstrated that these issues can be rapidly overcome via rational design and fragment assembly.

Key recent advances include:

(i) Sequence-based drug design across the human transcriptome to provide precision lead medicines

(ii) Small molecule cleavage of RNAs (RIBOTACS) in a catalytic and sub-stoichiometric manner via recruitment of cellular nucleases

(iii) Tools and technologies to study ligand binding capacity of RNAs across the transcriptome (Chem-CLIP and Ribo-SNAP)

(iv) Showing broad classes of known drugs target RNA and that their activity may be traced to targeting non-coding RNA

(v) Chemical biology approaches to understand RNA biology. We uncovered the mechanistic cause of Fragule X-Syndrome and Autism and also can define precisely the effect that non-coding RNAs have on the proteome.

(vi) Study druggability broadly. We have the ability to answer fundamental questions about how druggable the genome really is. Thus, we have launched the Druggable Transcriptome Project.

Publications:
Grants:
  • Oct 2025 ACTIVE
    Novel mRNA degraders to target hypoxic tumor microenvironment
    FL DEPT OF HLTH BANKHEAD-COLEY CANCER RE · Co-Investigator
  • Apr 2025 ACTIVE
    Targeted degradation of RNAs by using small molecules
    NATL INST OF HLTH NCI · Principal Investigator
  • Dec 2024 ACTIVE
    Small molecule degradation of tau mRNA
    RAINWATER CHARITABLE FOUNDATION · Principal Investigator
  • Jun 2024 – Dec 2025
    Developing Artificial Intelligence and Machine Learning Approaches to Identify and Optimize Small Molecule Medicines Targeting Oncogenic RNAs
    FL DEPT OF HLTH · Co-Project Director/Principal Investigator
  • Mar 2024 ACTIVE
    Design of small molecules that degrade the mRNA encoding a-synuclein
    SPARK NS · Principal Investigator
  • Dec 2023 – Nov 2024
    Brain penetrant small molecules drugs that target tau pre-mRNA for degradation
    RAINWATER CHARITABLE FOUNDATION · Principal Investigator
  • Oct 2023 – Oct 2025
    Astellas Pharma Inc. bench fees for Dr. Ryuichi Sekioka
    ASTELLAS PHARMA US · Principal Investigator
  • Sep 2023 – Aug 2025
    Graduate Research Fellowship program (GRFP)
    SCRIPPS RESEARCH INST · Principal Investigator
  • Sep 2023 ACTIVE
    Targeting the RNA Repeat Expansion that Causes Myotonic Dystrophy Type 2
    MUSCULAR DYSTROPHY ASSO · Principal Investigator
  • Sep 2023 ACTIVE
    Development of Clinical Candidates for the Treatment of Myotonic Dystrophy
    US ARMY MED RES ACQUISITION · Principal Investigator
  • Apr 2023 – Jun 2025
    CD33 Targeting Small Molecule Drug Discovery
    EISAI CO · Principal Investigator
  • Sep 2022 – Aug 2023
    Expanding Ribonuclease Targeting Chimeras (RIBOTACs) to Advance Targeted Degradation of Disease-Causing RNAs
    AMER CHEMICAL SOC · Other
  • Sep 2022 – Aug 2025
    Design and evaluation of drug-like small molecules targeting repeat expansion (MDA 963835)
    MUSCULAR DYSTROPHY ASSO · Other
  • May 2022 – Apr 2025
    Development of outpatient antiviral cocktails against SARS-CoV-2 and other potential pandemic RNA viruses
    STANFORD UNIV · Principal Investigator
  • May 2022 – Apr 2025
    Center for Antiviral Medicines & Pandemic Preparedness (CAMPP) – PROJECT 4
    SCRIPPS RESEARCH INST · Principal Investigator
  • Apr 2022 – Dec 2022
    Small Molecule Targeting of DUX4 mRNA for treatment of Fascioscapulohumeral Dystrophy (FSHD)
    PFIZER INC · Principal Investigator
  • Apr 2022 – Dec 2022
    Developing Pre-Clinical Candidates Against miRNAs Implicated in Autoimmune Diseases
    EMD SERONO RESEARCH & DEVELOPMENT INST · Principal Investigator
  • Apr 2022 – Apr 2023
    Pathophysiology of genetically defined dementia and neurodegeneration: Defining therapeutic targets and pathways
    NATL INST OF HLTH NINDS · Principal Investigator
  • Apr 2022 – May 2023
    RNA-Targeted Drug Discovery and Development for Parkinson's Disease
    RUTGERS STATE UNIV · Principal Investigator
  • Apr 2022 – Aug 2023
    Design and Study of Small Molecules That Cleave the RNA That Causes Myotonic Dystrophy Type 1 (DM1)
    US ARMY MED RES ACQUISITION · Principal Investigator
  • Apr 2022 – Oct 2023
    Targeting RNAs Associated with Tauopathies with Small Molecules
    RAINWATER CHARITABLE FOUNDATION · Principal Investigator
  • Apr 2022 – Oct 2023
    Pre-clinical evaluation of small molecules that rescue aberrant Tau mRNA splicing
    RAINWATER CHARITABLE FOUNDATION · Principal Investigator
  • Apr 2022 – Aug 2024
    Small Molecules That Target the RNAs That Cause Pulmonary Fibrosis and Polycystic Kidney Disease
    US ARMY MED RES ACQUISITION · Principal Investigator
  • Apr 2022 – Mar 2025
    Targeting mRNAs that Contribute to Neurological Disease
    EXPANSION THERAPEUTICS · Principal Investigator
  • Apr 2022 – Mar 2025
    Targeted degradation of RNAs by using small molecules
    NATL INST OF HLTH NCI · Principal Investigator
  • Apr 2022 – Jun 2025
    Small Molecule Targeting of mRNAs Encoding Proteins relevant for Cardiovascular, Renal and Metabolic Diseases
    ASTRAZENECA · Principal Investigator
  • Apr 2022 ACTIVE
    Design of precision small molecules targeting RNA repeating transcripts to manipulate and study disease biology
    NATL INST OF HLTH NINDS · Principal Investigator
Education:
  • 2003
    Ph.D. in Biophysical Chemistry
    University of Rochester
  • 1999
    Master's of Science in Chemistry
    University of Rochester
  • 1997
    Bachelor's of Science in Chemistry
    University of Maryland, College Park
Ezgi Hacisuleyman

Ezgi Hacisuleyman

Assistant Professor
Department: SR-MM-HACISULEYMAN LAB
Phone: (561) 228-2121
Physical Address:
Accomplishments:
  • Kavli Neuroscience Postdoctoral Fellowship
    2021-2023 · Kavli Neural Systems Institute at The Rockefeller University
  • Helen Hay Whitney Fellow
    2016-2019 · Helen Hay Whitney Foundation
Grants:
  • Aug 2025 ACTIVE
    Decoding RNA Localization and Local Translation: Key Mechanisms of Cellular Adaption and Function
    NATL INST OF HLTH NIGMS · Principal Investigator
Education:
  • 2010-2015
    Ph.D. in Molecular Biology and Biochemistry
    Harvard University
  • 2006-2010
    Bachelor of Science, Molecular Biology and Genetics
    Massachusetts Institute of Technology
  • 2006-2010
    Bachelor of Engineering, Chemical Engineering
    Massachusetts Institute of Technology
Michael Harris

Michael Harris

Professor
Department: LS-CHEMISTRY-GENERAL
Phone: (216) 212-5191
Mailing Address:
Grants:
  • Jul 2020 – Sep 2025
    Chemistry-Biology Interface Training Program at the University of Florida
    NATL INST OF HLTH NIGMS · Other
  • Jun 2020 – Jun 2022
    NMR console upgrade for structural biology and metabolomics
    NATL INST OF HLTH OD · Faculty
  • Jul 2018 – Jun 2025
    Specificity in Substrate Recognition and Catalysis by RNA Processing Enzymes
    NATL INST OF HLTH NIGMS · Principal Investigator
  • Mar 2017 – Feb 2019
    Mechanistic Enzymology of Phosphoryl Transfer Enzymes
    NATL INST OF HLTH NIGMS · Principal Investigator
Yanjun Li

Yanjun Li Ph.D.

Assistant Professor
Department: Medicinal Chemistry
Phone: (352) 273-9957
Mailing Address:
Physical Address:

Yanjun Li is an Assistant Professor (AI Initiative) in the Department of Medicinal Chemistry at the College of Pharmacy, University of Florida. His research interests span the fields of deep learning, drug discovery, and precision medicine, with a particular emphasis on AI-driven drug discovery. Dr. Li’s work aims to develop innovative AI algorithms to tackle foundational life science challenges with broad scientific impacts and to optimize and automate real-world drug discovery and design pipelines.

Dr. Li earned his Ph.D. in computer science from the University of Florida and then worked as a Senior Research Scientist at the Institute of Deep Learning, Baidu Research USA in Sunnyvale, California. His passion for real-world drug discovery led him to join Calico Life Sciences, an Alphabet-founded research and development lab for human aging research, as a Machine Learning Scientist. In 2023, Dr. Li started his tenure-track Assistant Professorship in the Department of Medicinal Chemistry at the University of Florida, where he continues to pursue cutting-edge research in AI-driven drug discovery.

Accomplishments:
  • UF Health Cancer Center Annual Research Showcase Poster Winner
    2025 · University of Florida Health Cancer Institute
  • UF College of Pharmacy 38th Annual Research Showcase Poster Winner,
    2025 · College of Pharmacy, University of Florida
  • Excellence in Instructional Redesign
    2025 · College of Pharmacy, University of Florida
Research Summary:

Dr. Li’s research focuses on AI-driven drug discovery and optimization, utilizing deep learning techniques to accelerate the discovery of novel functional molecules for improved human health outcomes. His current research interests include: (1) developing deep learning algorithms for the design of small molecules, peptides, antibodies, and domain proteins to expedite the drug design pipeline and lead to potential drug candidates; (2) predicting molecular interactions through domain-tailored deep learning methods and molecular dynamics simulations; (3) exploring molecular structure-property relationships to guide the structure-based drug design and optimization; (4) analyzing multi-omics data, electronic health records, and medical imaging data for drug repurposing and synergistic combinations with machine learning methods; (5) advancing the capacity of machines to understand the intricate language of chemistry with natural language processing.

Dr. Li’s laboratory fosters a culture of interdisciplinary and translational collaboration to cultivate a dynamic and distinctive working environment. The lab endeavors to advance the frontiers of AI-driven drug discovery while propelling the field of artificial intelligence towards real-world applications in the life sciences.

Publications:
Grants:
  • Aug 2025 ACTIVE
    Cell Morphology-guided, Scalable and Controllable Molecule Design via Generative AI
    NATL INST OF HLTH NIBIB · Principal Investigator
  • Aug 2024 – Aug 2025
    Hijacking Post-translational Arginylation for Targeted Protein Degradation
    WASHINGTON UNIV SAINT LOUIS · Principal Investigator
  • Jun 2024 – Dec 2025
    Developing Artificial Intelligence and Machine Learning Approaches to Identify and Optimize Small Molecule Medicines Targeting Oncogenic RNAs
    FL DEPT OF HLTH · Co-Project Director/Principal Investigator
  • Jun 2024 – Dec 2025
    Enhancing Cancer Diagnosis and Treatment through Innovative Glycan- Specific Protein Engineering
    FL DEPT OF HLTH · Co-Investigator
  • Dec 2023 – Dec 2024
    ExoTarget Platform as a programmable delivery system
    NATL INST OF HLTH · Co-Investigator
  • Jun 2023 ACTIVE
    UF Health Cancer Center Support Grant
    NATL INST OF HLTH NCI · Project Manager
Education:
  • 2021
    Ph.D. (Computer Science)
    University of Florida
  • 2015
    M.S. (Information, Production and Systems Engineering)
    Waseda University
  • 2015
    M.S. (Communication and Information System)
    University of Electronic Science and Technology of China
  • 2012
    B.S. (Information Engineering)
    University of Electronic Science and Technology of China
Chenglong Li

Chenglong Li Ph.D.

Chair of Medicinal Chemistry and the Nicholas Bodor Professor in Drug Discovery
Department: Medicinal Chemistry
Phone: (352) 294-8510
Email: lic@ufl.edu
Mailing Address:
Physical Address:

Chenglong Li, Ph.D., obtained his B.Sc. in chemistry and M.Sc. in physical chemistry from Beijing University in 1985 and 1988, respectively. Following college, he worked at the Institute of Biophysics at the Chinese Academy of Sciences. He came to the United States and obtained his Ph.D. in biophysics at Cornell University, New York in 2000. He spent five years in San Diego, California, working as a postdoc in structural biology at the Burnham Institute for Medical Research (2000-2002) and as a research associate in computational chemistry at the Scripps Research Institute (TSRI, 2002-2005). Starting August 1, 2005, he moved to Columbus, Ohio as a tenure-track assistant professor in the Division of Medicinal Chemistry and Pharmacognosy at the College of Pharmacy, the Ohio State University, and rose through the ranks to associate professor with tenure in 2011 and full professor in 2016. In August 2016, he was appointed as the Nicholas Bodor Professor in Drug Discovery and professor of medicinal chemistry at the University of Florida. In 2026, he was named chair of the Department of Medicinal Chemistry.

Research Summary:

Li’s scholarly interests range from organic chemistry, biochemistry, medicinal chemistry to physical chemistry, computational chemistry, molecular biophysics and pharmacology. His research focuses on molecular recognition, with a strong application to structure-based computer-aided drug design. He combines molecular simulation, synthetic chemistry, X-ray protein crystallography, thermodynamic measurements, cellular techniques and in vivo animal models to explore molecular interactions, especially protein-ligand interactions, at molecular, cellular and organismal levels. His current working projects include both computational method development and drug design applications, for example: 1) pioneering development of a novel Multiple Ligand Simultaneous Docking (MLSD) strategy, with great potential for Fragment-Based Drug Design (FBDD); 2) design and discovery of drugs targeting the IL-6/STAT3 inflammatory and oncogenic pathway for targeted therapy; 3) design and discovery of drugs targeting epigenetic histone arginine methylation enzymes, especially PRMT5; 4) design and discovery of drugs targeting specific nAChR and ASIC1 subtypes for drug addiction and neurodegenerative diseases; 5) design and discovery of “chemical chaperone” drugs targeting F508 NBD1 misfolding intermediates for potential cystic fibrosis therapy.

• h-index: https://scholar.google.com/citations?user=cpPZNU0AAAAJ&hl=en&oi=ao • First-in-Class PRMT5-targeting epigenetic drug commercialization development licensing agreement • NIH DDNS (Drug Discovery for the Nervous System) study section regular membership

Publications:
Grants:
  • Sep 2024 – Aug 2025
    AI-directed discovery of heparanase inhibitors
    NATL INST OF HLTH NIDDK · Principal Investigator
  • Jul 2024 ACTIVE
    Dual Inhibition of PARP and IL-6 as a Novel Therapeutic Approach for BRCA-Mutated Triple-Negative Breast Cancer
    US ARMY MED RES ACQUISITION · Principal Investigator
  • Jun 2024 – Dec 2025
    Developing Artificial Intelligence and Machine Learning Approaches to Identify and Optimize Small Molecule Medicines Targeting Oncogenic RNAs
    FL DEPT OF HLTH · Co-Project Director/Principal Investigator
  • Feb 2024 – Jan 2026
    Dual inhibition of PARP and STAT3 as a novel therapeutic approach for triple-negative breast cancer
    UNIV OF MARYLAND BALTIMORE · Principal Investigator
  • Sep 2023 ACTIVE
    Mechanisms of Metal Ion Homeostasis of Oral Streptococci
    NATL INST OF HLTH NIDCR · Co-Investigator
  • Jul 2023 ACTIVE
    Developing YAP/TEAD Inhibitors for Novel Nash-Associated Hepatocellular Carcinoma Therapeutics
    US ARMY MED RES ACQUISITION · Principal Investigator
  • May 2023 ACTIVE
    Development of high-affinity and selective ligands as a pharmacological tool for the Dopamine D4 receptor (D4R) subtype variants
    HIGH POINT UNIVERSITY · Principal Investigator
  • Aug 2022 ACTIVE
    Integrative Multidisciplinary Discovery Platform to Unlock Marine Natural Products Therapeutic Opportunities
    NATL INST OF HLTH NIGMS · Co-Investigator
  • Feb 2022 – Jan 2025
    Computationally designed IL-10 mutants for the treatment of inflammatory bowel diseases
    US ARMY MED RES ACQUISITION · Co-Investigator
  • Jan 2022 – Jan 2023
    HEC Bench Fee – Tahira Noor
    HIGHER EDUCATION COMMISSION PAKISTAN · Principal Investigator
  • Jan 2021 – Dec 2022
    Artificial Intelligence Research Catalyst Fund
    UF RESEARCH · Project Manager
  • Jul 2020 – Sep 2025
    Chemistry-Biology Interface Training Program at the University of Florida
    NATL INST OF HLTH NIGMS · Principal Investigator
  • Sep 2019 – Aug 2023
    QLCI-CG: Conceptualization of the Institute for Quantum Biology on Quantum Computers
    NATL SCIENCE FOU · Co-Project Director/Principal Investigator
  • Sep 2019 – Jan 2025
    Novel Targeted Anticancer Agents from Marine Cyanobacteria
    NATL INST OF HLTH NCI · Other
  • Sep 2018 – Aug 2022
    Mechanistic studies of gamma-glutamyl transpeptidase inhibition: A novel approach to modulating serum levels of cysteine
    UNIV OF OKLAHOMA NORMAN · Principal Investigator
  • Jun 2017 – May 2023
    Role and targeting of PRMT5 in prostate cancer
    PURDUE UNIV · Principal Investigator
  • Sep 2016 – Jan 2018
    Repositioning Bazedoxifene as a novel IL-6/GP130 inhibitor for sarcoma therapy
    UNIV OF MARYLAND COLLEGE PARK · Principal Investigator
  • Aug 2016 – Apr 2019
    Small molecule in vivo probe development targeting the IL-6/STAT3 pathway for potential multiple sclerosis therapy
    NATL INST OF HLTH NINDS · Principal Investigator
  • Aug 2016 – May 2021
    A novel STAT3-selective inhibitor for medulloblastoma therapy
    UNIV OF MARYLAND COLLEGE PARK · Principal Investigator
Patents:
  • Published February 2019
    IL-6 Inhibitors and Methods of Treatment
    #US-2021-0206718-A1
Education:
  • 2000
    Ph.D.
    Cornell University
  • 1988
    M.Sc.
    Beijing University
  • 1985
    B.Sc.
    Beijing University
Zhonglin Liu

Zhonglin Liu

AST PROF
Department: SR-CHEM-LIU LAB
Phone: (561) 228-2000
Physical Address:
Research Summary:

Liu lab focuses on developing new chemical modalities, chemical probes, and ABPP platforms to discover novel therapeutic approaches for human diseases.

Research Interests:
  • Chemical biology
  • Genetics and Genomics
  • Medicinal chemistry
  • Molecular Biology
  • proteomics
Publications:
Grants:
  • Jun 2023 ACTIVE
    UF Health Cancer Center Support Grant
    NATL INST OF HLTH NCI · Project Manager
Education:
  • 2012-2017
    Ph.D.
    Shanghai Institute of Organic Chemistry, CAS
  • 2008-2012
    B.S.
    Nanchang University
Sathyanarayanan V Puthanveettil

Sathyanarayanan V Puthanveettil Ph.D.

Associate Professor
Department: SR-NEURO-PUTHANVEETTIL LAB
Phone: (561) 228-2243
Mailing Address:

Dr. Puthanveettil graduated from Washington State University where he studied calcium calmodulin dependent protein kinases in the laboratory of Dr. B. W. Poovaiah. He then joined Dr. Eric Kandel’s laboratory at the Howard Hughes Medical Institute, Columbia University, for his post-doctoral research on learning and memory storage.

Accomplishments:
  • CAREER Award
    2015 · National Science Foundation
  • Whitehall Foundation Award
    2012 · Whitehall Foundation
  • Young Investigator Award
    2011 · Margaret Q. Landenberger Research Foundation
  • TSRI Nomineee for Alfred Sloan Award, Searle Scholar Award
    2011 ·
  • Columbia University Nominee for Blavatnik Award
    2010 · New York Academy of Sciences
  • Recognition for work on Kinesin transported RNAs as “HOT TOPIC” for Neuroscience 2009
    2009 · Society For Neuroscience (SFN)
  • Cold Spring Harbor Laboratory Registration Waiver
    2009 ·
  • Prize for Innovative Research Ideas
    2003 · The National Neurofibromatosis Foundation and International Neurofibromatosis Research Foundation
  • Travel Grant
    2000 · American Society for Gravitational and Space Biology (ASGSB)
  • Travel Award
    2000 · Washington State University (WSU) Graduate School
  • Gordon Research Conference Chair’s Funds for Registration Fees
    2000 ·
  • Cold Spring Harbor Laboratory Registration waiver
    1997 ·
  • National Merit Scholarship
    1984-1991 · Department of Education, India
Research Summary:

Molecular Logic For The Storage and Maintenance Of Long-Term Memory

The long-term goal of my laboratory is to understand the molecular and cellular basis of memory storage and cognitive disorders. At the cellular level there are two hallmarks of long-term memory storage: experience-dependent activation of gene expression in the nucleus, and local activation of translation at specific synapses.

Previously I have demonstrated in Eric Kandel’s laboratory that activation of fast axonal transport in pre- and post- synaptic neurons during learning is another critical component of long-term memory storage. Activation of molecular transport to synapses is a rate limiting step and coordinates nuclear and synaptic processes during memory storage. The transported cargos include organelles, proteins, mRNAs, and noncoding RNAs.

In an effort to learn what gene products are transported in specific neurons in response to activity, how they are transported, how they are stored for later use, and eventually when and how they are utilized at specific synapses during learning and memory storage, my laboratory uses an integrated approach that combines several high throughput techniques such as genomics and proteomics with electrophysiology, biochemistry, and imaging. A comprehensive understanding of transported cargos will help us in elucidating signaling pathways at the synapse and mechanisms underlying synaptic dysfunction. Animal models such as Aplysia and mice are used to tackle these questions at the cellular and systems levels. In addition, we explore these questions using mouse models for cognitive disorders such as Alzheimer’s and Huntington’s disease.

Publications:
Grants:
  • May 2025 ACTIVE
    Determining the Molecular Arrangement of GABABR-GIRK Signaling Cascades
    NATL INST OF HLTH NIDA · Other
  • Mar 2025 – Oct 2025
    Defining the mechanism of PTCHD1 and membrane cholesterol in the regulation of the mu-opioid receptor
    NATL INST OF HLTH NIGMS · Other
  • Jan 2025 ACTIVE
    Cell specific & compartment specific changes in translation during long term memory storage
    NATL INST OF HLTH NIMH · Other
  • Jan 2024 – Jan 2025
    Molecular basis for cholesterol regulation of Kir2.1 in cardiac arrhythmia
    AMER HEART ASSOCIATION · Other
  • Dec 2023 – Nov 2025
    Analysis of RNAs targeted to the long-distance projections of ventral hippocampus
    NATL INST OF HLTH NIMH · Principal Investigator
  • Sep 2022 – Aug 2024
    Novel role of mitotic kinesin KIF11 in structural plasticity and memory
    NATL INST OF HLTH NIMH · Other
  • Jun 2022 – May 2025
    Assessing effects of manipulation of expression of kinesins in a mouse model of Alzheimer's disease
    NATL INST OF HLTH NIA · Principal Investigator
  • Apr 2022 – Jun 2023
    Assessing the role of circRNAs in memory consolidation
    NATL INST OF HLTH NIMH · Principal Investigator
  • Apr 2022 – Aug 2024
    The role of bidirectional transport of lysosome-related organelles in learning and memory storage
    NATL INST OF HLTH NIMH · Principal Investigator
  • Apr 2022 – Oct 2024
    Dynein-Dynactin Complex Mediated Retrograde Transport during Long-term Memory Storage
    NATL INST OF HLTH NIMH · Principal Investigator
  • Apr 2022 – Jul 2025
    Transcriptomic Mechanisms of Formation and Persistence of Synapse Specific Long-Term Memory
    NATL INST OF HLTH NIMH · Principal Investigator
  • Apr 2022 – Feb 2025
    Bidirectional Transport of Lysosome Related Organelles during Synapse Maintenance and Plasticity
    NATL SCIENCE FOU · Principal Investigator
Education:
  • 2001
    Ph.D. in Biochemistry/Biophysics
    Washington State University
  • 1994
    Master's of Science in Biotechnology
    Tamil Nadu Agricultural University
  • 1991
    Bachelor's of Science in Agriculture
    Kerala Agricultural University
Laura P W Ranum

Laura P W Ranum

Kitzman Family Professor Of Molecular Genetics And Microbiology; Director Center For NeuroGenetics
Department: Molecular Genetics & Microbiology
Phone: (352) 294-5209
Email: ranum@ufl.edu
Mailing Address:
Physical Address:

Fun Fact: I love to hike and my favorite hike is the double tea house hike near Lake Louise, Canada. Our lab focuses on the role of Repeat Associated Non-ATG (RAN) translation, RNA gain of function and protein gain of function in repeat expansion disorders including amyotrophic lateral sclerosis (ALS), spinocerebellar ataxia (SCA) types 5 and 8, myotonic dystrophy (DM) types 1 and 2, and Huntington’s disease (HD). Additionally, we continue to search for novel human disease genes. We are using high-throughput sequencing strategies to look for single-gene mutations that cause novel forms of ataxia, ALS, and neuropsychiatric diseases. Our scientific breakthroughs over the years have depended on partnerships with talented colleagues and students and with members of the community who participated in our research studies. Although much work remains, scientific advances have dramatically increased opportunities for drug development and clinical trials. Our goal is to perform cutting-edge research that will lead to improvements in diagnosis and treatments for neurological and neuromuscular disease.

Research Interests:
  • Amyotrophic Lateral Sclerosis
  • Gene Discovery
  • Genetics of Neurodegenerative Disease
  • Multiple System Atrophy (MSA)
  • Myotonic Dystrophy
  • Neurodegenerative diseases
  • Neuropathology of Neurodegenerative diseases
  • Repeat Expansion Diseases
  • Spinocerebellar Ataxia
Publications:
Grants:
  • Sep 2025 ACTIVE
    Molecular effects of metformin, PKR and TBI on C9orf72 ALS/FTD
    NATL INST OF HLTH NINDS · Principal Investigator
  • Jul 2025 ACTIVE
    Novel repeat associated non-AUG (RAN) proteins in sALS, sFTD and SBMA: shared pathological features and unifying therapeutic opportunities
    NATL INST OF HLTH NINDS · Principal Investigator
  • May 2025 ACTIVE
    Proof of concept testing of an anti-GA immunotherapy for C9orf72 ALS/FTD
    LATUS BIO · Principal Investigator
  • Dec 2024 – Nov 2025
    10th International Unstable Microsatellites and Human Disease Meeting
    NATL INST OF HLTH NINDS · Principal Investigator
  • Aug 2024 ACTIVE
    Senator Paul D. Wellstone Muscular Dystrophy Specialized Research Center
    NATL INST OF HLTH NINDS · Faculty
  • Apr 2024 ACTIVE
    Contribution of Repeat Associated Non-AUG (RAN) Proteins in Friedreich's and Other Hereditary Ataxias
    US ARMY MED RES ACQUISITION · Principal Investigator
  • Sep 2023 ACTIVE
    Safety of Metformin in C9orf72 ALS: Effects on RAN Proteins, Breathing, Imaging, and Metabolomic Outcome Measures
    US ARMY MED RES ACQUISITION · Principal Investigator
  • Sep 2023 ACTIVE
    Understanding and targeting common mechanisms of myotonic dystrophy types 1 and 2 using novel repeat expansion mouse models
    US ARMY MED RES ACQUISITION · Principal Investigator
  • May 2023 ACTIVE
    Anti-RAN targeting immunotherapy for C9orf72 ALS and genetically unknown RAN-positive sporadic ALS
    US ARMY MED RES ACQUISITION · Principal Investigator
  • Mar 2023 – Feb 2025
    Repeat associated non-AUG (RAN) proteins in FA: contribution to disease and therapeutic opportunities
    FRIEDREICHS ATAXIA RESEARCH ALLIANCE · Other
  • Feb 2023 ACTIVE
    An RNA-based nanoparticle vaccine approach for the prevention and treatment of C9orf72 ALS/FTD
    US ARMY MED RES ACQUISITION · Principal Investigator
  • Dec 2022 – Nov 2024
    Safety & therapeutic potential of metformin for C9orf72 ALS
    AMYOTROPHIC LATERAL SCLEROSIS ASSOC · Principal Investigator
  • Sep 2022 – Aug 2025
    Molecular effects of metformin, PKR and TBI on C9orf72 ALS/FTD
    NATL INST OF HLTH NINDS · Principal Investigator
  • Jul 2022 ACTIVE
    Identifying and targeting novel repeat associated non-AUG (RAN) proteins in sporadic ALS
    US ARMY MED RES ACQUISITION · Principal Investigator
  • Jul 2022 – Jun 2025
    Novel repeat associated non-AUG (RAN) proteins in sALS, sFTD and SBMA: shared pathological features and unifying therapeutic opportunities
    NATL INST OF HLTH NINDS · Principal Investigator
  • Jan 2022 – Dec 2023
    Characterization of a Novel BAC Transgenic Mouse Model for DM2
    MYOTONIC DYSTROPHY FOU · Other
  • Aug 2021 – Jul 2023
    Using mitochondrial Ca2+ uptake as a therapeutic target for ALS
    NATL INST OF HLTH NINDS · Co-Investigator
  • Jul 2021 ACTIVE
    Contribution of RAN proteins to HD, SCA3 other CAG.CTG expansion diseases
    NATL INST OF HLTH NINDS · Principal Investigator
  • Mar 2021 – Feb 2022
    RAN proteins in sporadic ataxias and SCA8: diagnostic tools and therapeutic potential of metformin
    NATL ATAXIA FOUNDATION · Other
  • Sep 2020 – Jun 2023
    Identifying and understanding the role of repeat RNAs and RAN proteins in Alzheimer's disease
    NATL INST OF HLTH NIA · Other
  • Apr 2020 – Mar 2021
    An endobody vaccine against a combination of RAN proteins as a therapeutic approach in a C9 ALS BAC mouse model
    JOHNS HOPKINS UNIVERSITY · Principal Investigator
  • Jan 2020 – Jan 2023
    Provenance Initiatives Fund at the Center for NeuroGenetics
    UF FOUNDATION · Principal Investigator
  • Nov 2019 – Oct 2021
    C9ORF72 ALS: Endobody vaccine for the treatment of C9 ALS/FTD
    AMYOTROPHIC LATERAL SCLEROSIS ASSOC · Other
  • Sep 2019 – Sep 2022
    Therapeutic Potential of Metformin, an FDA-approved drug, and PKR Targeting for C9orf72 ALS
    US ARMY MED RES AND DEVELOPMENT COMMAND · Principal Investigator
  • Dec 2018 – Jan 2021
    Presymptomatic biomarkers of C9-ALS/FTD
    AMYOTROPHIC LATERAL SCLEROSIS ASSOC · Principal Investigator
  • Sep 2018 – Apr 2020
    Assessment of endobody vaccine for effectiveness and ability to generate C9 RAN antibodies
    UNITED NEUROSCIENCE LIMITED · Principal Investigator
  • Aug 2018 – Feb 2021
    C9ORF72 ALS: Proof of biology for antibody treatment in C9-ALS mouse model
    BIOGEN MA INC · Principal Investigator
  • Aug 2018 – Jul 2021
    The contribution of RAN proteins to C9ORF72 ALS/FTD
    MUSCULAR DYSTROPHY ASSO · Principal Investigator
  • Feb 2018 – Feb 2020
    Therapeutic potential of CRISPR-Cas9 in C9ORF72-repeat deletion strategy in C9ORF72 BAC transgenic mice
    CRISPR THERAPEUTICS AG · Principal Investigator
  • Jan 2018 – Dec 2019
    Molecular characterization of RNA and RAN protein effects in DM2
    WYCK FOUNDATION · Other
  • Jul 2017 – Jun 2022
    Neurodegenerative disease research
    UF FOUNDATION · Principal Investigator
  • Jul 2017 – Dec 2024
    University of Florida/National Ataxia Foundation Brain Donation Program
    NATL ATAXIA FOUNDATION · Principal Investigator
  • May 2017 – Dec 2020
    Therapeutic potential of CRISPR-Cas9 in C9ORF72 ALS/FTD-repeat deletion and transcriptional repression strategies in humanized C9ORF72 BAC transgenic mice
    TARGET ALS FOUNDATION · Principal Investigator
  • Jan 2017 – Jun 2020
    Nuclear Export Inhibitor KPT-350 for C9orf72 and Sporadic ALS
    TARGET ALS FOUNDATION · Principal Investigator
  • Jan 2017 – Jun 2020
    Developing human-derived antibodies to target dipeptide-repeat protein toxicity in C9orf72 disease
    TARGET ALS FOUNDATION · Principal Investigator
  • Aug 2016 – Oct 2017
    Developing a high-throughput screening assay for identification of small molecules that selectively target transcription of G4C2/G2C4 repeat expansions in the gene C9ORF72 in ALS
    AMYOTROPHIC LATERAL SCLEROSIS ASSOC · Other
  • Aug 2016 – Jul 2018
    Molecular characterization & antibody therapy in a novel C9orf72 BAC mouse model
    MUSCULAR DYSTROPHY ASSO · Principal Investigator
  • Aug 2016 – Jun 2022
    Molecular Characterization of ALS/FTD in a novel C9orf72 BAC mouse model.
    NATL INST OF HLTH NINDS · Principal Investigator
  • Mar 2016 – Mar 2018
    The Therapeutic Efficacy of Neurimmunes Human Derived Recombinant Antibodies in C9orf72 Transgenic Mouse Models
    NEURIMMUNE AG · Principal Investigator
  • Mar 2016 – Apr 2021
    neurodegenerative disease research
    UF FOUNDATION · Principal Investigator
  • Jan 2016 – Jul 2022
    Molecular Genetics of the G4C2 expansion mutation in ALS/FTD
    UF FOUNDATION · Principal Investigator
  • Jan 2016 – Jun 2023
    Molecular genetic characterization of SCA8
    UF FOUNDATION · Principal Investigator
  • Jan 2016 – Dec 2016
    ASO targeting of bidirectional transcripts and RAN translation in SCA8
    NATL ATAXIA FOUNDATION · Principal Investigator
  • Dec 2015 – Dec 2020
    Brainstorming microsatellite expansion diseases symposium
    UF FOUNDATION · Principal Investigator
  • Aug 2015 – Jul 2023
    Molecular Genetic Characterization of SCA8
    NATL INST OF HLTH NINDS · Principal Investigator
  • Jan 2015 – Jan 2018
    MBI Research Program MGOLD1
    UF FOUNDATION · Principal Investigator
  • Sep 2013 – Jun 2019
    Myotonic Dystrophy: Molecular Pathophysiology and CNS Effects
    NATL INST OF HLTH NINDS · Principal Investigator
  • Jul 2013 – Jun 2019
    Matching Funds for PPG Project Titled: "Myotonic Dystrophy: Molecular Pathophysiology and CNS Effects"
    FL CLINICAL PRACTICE ASSO · Principal Investigator
  • Jul 2013 – Jun 2023
    Center for NeuroGenetics
    UF FOUNDATION · Principal Investigator
  • May 2013 – Apr 2016
    Repeat associated non-ATG (RAN) Translation in C9ORF72 ALS/FTD
    COLUMBIA UNIVERSITY · Principal Investigator
Patents:
  • Published April 2023
    Methods for Diagnosing Huntington's Disease
    #US-2023-0288434-A1
  • Published May 2022
    Regulation of RAN Translation by PKR and eIF2a-P Pathways
    #US-2023-0002753-A1
  • Published March 2022
    Vaccine Therapy for RAN Protein Diseases
    #US-2023-0218730-A1
  • Issued June 2021
    Use and Treatment of Di-Amino Acid Repeat-Containing Proteins Associated with ALS
    #11,035,867
  • Published May 2021
    Use and Treatment of Di-Amino Acid Repeat-Containing Proteins Associated with ALS
    #US-2021-0285970-A1
  • Issued March 2021
    Manipulation of eIF3 to Modulate Repeat Associated Non-ATG (RAN) Translation
    #10,940,161
  • Published January 2021
    Manipulation of eIF3 to Modulate Repeat Associated Non-ATG (RAN) Translation
    #US-2021-0236535-A1
  • Issued May 2020
    Use and Treatment of Di-Amino Acid Repeat-Containing Proteins Associated with ALS
    #10,663,475
  • Issued December 2019
    Methods for Diagnosing Huntington's Disease
    #10,509,045
  • Published November 2019
    Methods for Diagnosing Huntington's Disease
    #US20/0241013
  • Issued May 2019
    Use and Treatment of Di-Amino Acid Repeat-Containing Proteins Associated with ALS
    #10,295,547
  • Published September 2018
    Use of Metformin and Analogs Thereof to Reduce RAN Protein Levels in the Treatment of Neurological Disorders
    #US-2020-0268691-A1
  • Published September 2018
    MSD Assay to Detect Dipeptide RAN Proteins in C9orf72 ALS/FTD
    #US20/02392925
  • Published April 2018
    Regulation of RAN Translation by PKR and eIF2a-P Pathways
    #US-2020-0140846
Rolf Renne

Rolf Renne

Associate Director for Basic Science, Henry E. Innes Professor of Cancer Research
Department: Molecular Genetics & Microbiology
Phone: (352) 273-8204
Mailing Address:
Physical Address:

Rolf Renne, Ph.D., is a Professor in the Department of Molecular Genetics and Microbiology, and has studiesd different aspects of Kaposi’s sarcoma-associated herpesvirus (KSHV) since 1995. His current focus is on epigenetic regulation of viral latency and the role of long and small non-coding RNAs in viral biology. His laboratory is using genomics, genetics, and ribonomics approaches to study how viral-encoded genes and microRNAs contribute to KSHV biology and tumorigenesis.

Dr. Renne received his Master’s (1989) and Ph.D. (1993) degrees from the Albert-Ludwigs University of Freiburg, Germany. He performed most of his Ph.D. thesis work at the University of California Davis with Dr. Paul Luciw in the field of Retrovirology. Next, he joined the laboratory of Dr. Don Ganem, UCSF, where he contributed to early work on establishing tissue culture models for KSHV. In 1999, he joined the faculty at Case Western Reserve University, where he focused on molecular aspects such as DNA binding and DNA replication of the KSHV latency-associated nuclear antigen (LANA) and the establishment of the only available KS xenograft model. In 2004, he moved to the University of Florida, where his laboratory was one of four to identify KSHV-encoded microRNAs. More recently, Dr. Renne has contributed to studies on HSV-1, EBV, HHV-6, MHV-68, and poxviruses. Dr. Renne has been continuously funded by multiple NCI grants since 2004, and in 2017, was awarded a multi-investigator NCI Program project together with Drs. Scott Tibbetts (UF), and Erik Flemington (Tulane University). This P01 Project was successfully renewed in 2022. Dr. Renne is on the editorial board of Journal of Virology, Virology, and serves on multiple NIH special emphasis panels. In 2009, he co-chaired the 11th International Workshop on KSHV and related agents and in 2015 the 40th International Herpesvirus Workshop. The unifying theme of his research projects is to deepen our understanding on how latent gamma-herpesvirus gene products including microRNAs and long noncoding RNAs contribute to viral tumorigenesis. While his focus is on Kaposi’s sarcoma, Primary effusion lymphomas, he also collaborates on EBV-associated cancer, and animal models such as MHV68 and various noninfectious cancers including breast cancer and melanoma. In 2017, he was appointed Associate Director for Basic Science at the UF Health Cancer Institute, and in 2019 was named the Henry E. Innes Professor of Cancer Research. In 2021, he was appointed member of the NCI panel A for Cancer Center reviews.

Research Summary:

Fun Fact: My dream is to sail around the world one day. The long-term goal of our research is to understand how latency-associated genes of Kaposi’s Sarcoma-associated Herpesvirus (KSHV), including short (microRNAs) and long noncoding RNAs contribute to viral latency and tumorigenesis. KSHV causes Kaposi’s sarcoma and multiple B cell malignancies in immunosuppressed patients. In South Saharan Africa KS is the most prevalent cancer in man. KSHV has been discovered nearly 30 years ago, however to this date we have not been able to develop an efficient therapeutic approach that targets the virus directly. Studying KSHV is complicated by the fact that we have no practical animal models to study pathogenesis. Hence, to decipher the role of virally encoded long and short noncoding RNAs in tumorigenesis, we have created a highly collaborative approach to comparatively study noncoding RNAs in KSHV, Epstein-Barr Virus, the other human gamma-herpesvirus associated with cancer in humans (with Dr. Flemington at Tulane) and MHV-68 a natural Mouse gamma-herpesvirus associated with B cell lymphomas in mice (with Dr. Scott Tibbetts, MGM UF). Using genetics, genomics, proteomics, and ribonomics-based approaches, we have identified a number of pathways and molecular mechanisms of how noncoding RNAs contribute to pathogenesis of all three viruses. The long-term goal is to identify viral RNAs expressed by the virus or host RNAs perturbed by viral infection that represent tumor vulnerabilities and can be therapeutically targeted

Publications:
Grants:
  • Jun 2025 ACTIVE
    The Florida Summer Training in Research and Oncology for the Next Generation of Researchers (STRONGER) Program
    NATL INST OF HLTH NCI · Other
  • Jun 2023 ACTIVE
    UF Health Cancer Center Support Grant
    NATL INST OF HLTH NCI · Other
  • Aug 2022 ACTIVE
    Investigating miR-9-5p and its targets as interventional and clinicopathologic predictors of laryngeal cancer racial disparate outcomes
    US ARMY MED RES ACQUISITION · Other
  • Jun 2022 ACTIVE
    Noncoding RNAs in gamma-Herpesvirus Biology and AIDS Malignancies
    NATL INST OF HLTH NCI · Principal Investigator
  • Jan 2022 ACTIVE
    MicroRNA turnover induced by target RNAs in Colorectal Cancer
    AMERICAN CANCER SOCIETY · Co-Investigator
  • Jan 2019 – Oct 2020
    UFF F006200 Innes Professorship
    UF FOUNDATION · Principal Investigator
  • Sep 2018 – Jun 2024
    The Role of H3.3 histone variant in the pathogenesis of oral Kaposi's Sarcoma
    NATL INST OF HLTH NIDCR · Principal Investigator
  • Feb 2017 – Jan 2022
    Noncoding RNAs in gamma-Herpesvirus Biology and AIDS Malignancies
    NATL INST OF HLTH NCI · Principal Investigator
  • Jul 2015 – Jun 2024
    Florida Academic Cancer Center Alliance Seed Grants
    UF HEALTH SHANDS HOSPITAL · Project Manager
  • Feb 2015 – Oct 2023
    UF Health Cancer Center Pilot Project Grants funded through the Florida Consortium of National Cancer Institute Centers Program
    UF HEALTH SHANDS HOSPITAL · Project Manager
  • Jan 2015 – Dec 2020
    Role of MHV68 miRNAs in latency and pathogenesis
    NATL INST OF HLTH NIAID · Co-Investigator
  • Jan 2015 – May 2024
    UF Health Cancer Center Bridge Seed Grant funded through the Florida Consortium of National Cancer Institute Centers Program
    UF HEALTH SHANDS HOSPITAL · Project Manager
  • Jul 2014 – Jun 2017
    Histone variant H3.3 and KSHV LANA in the pathogenesis of oral Kaposis sarcoma
    NATL INST OF HLTH NIDCR · Principal Investigator
  • Aug 2013 – Aug 2016
    Identification of KSHV miRNA targets in endothelial cells and characterization miRNA knockout viruses
    NATL INST OF HLTH · Principal Investigator
  • Dec 2012 – Jan 2019
    Studying the role of KSHV-encoded miRNAs
    NATL INST OF HLTH NCI · Principal Investigator
  • Mar 2011 – Feb 2017
    LANA and Cellular Gene Expression
    NATL INST OF HLTH NCI · Principal Investigator
  • Mar 2006 – May 2023
    Davis Cancer Researach Equipment
    UF FOUNDATION · Project Manager
Education:
  • 1989-1993
    PhD, Retrovirology/Foamy Viruses
    Albert-Ludwigs University
  • 1984-1989
    MS, Biology
    Albert-Ludwigs University
Elias Sayour

Elias Sayour MD, PhD

Stop Children’s Cancer/Bonnie R. Freeman Professor for Pediatric Oncology Research; Assistant Dean
Department: MD-NEUROLOGICAL SURGERY
Phone: (352) 273-9000
Mailing Address:
Physical Address:

Elias Sayour, MD, PhD, is the Stop Children’s Cancer/Bonnie R. Freeman Professor for Pediatric Oncology Research in the UF departments of neurosurgery and pediatrics at the University of Florida. He is also a principal investigator of the ribonucleic acid engineering laboratory at the Preston A. Wells, Jr. Center for Brain Tumor Therapy. He received his bachelor’s degree from Fordham University, his medical degree from the University of Buffalo and his doctorate from Duke University.

He completed his residency in pediatrics at Cohen’s Children’s Medical Center in New York and his fellowship at Duke University Medical Center. During his fellowship training, he completed a two-year National Institutes of Health research fellowship in cancer biology and developmental therapeutics. His primary research focus is developing tumor RNA loaded nanocarriers to re-direct host immunity against pediatric brain tumors.

Dr. Sayour is an NIH-funded investigator focused on on developing new nanotech vaccines to reprogram the immune system against cancer cells. He is investigating the use of personalized nanoparticles small enough to deliver essential information to the immune system educating it reject pediatric cancer. Currently his group is investigating the safety and efficacy of this novel vaccine formulation in canines with malignant brain tumors before translation into dedicated human studies.

Dr. Sayour’s work has been nationally recognized by the American Society of Pediatric Hematology-Oncology, National Institutes of Health, and U.S. Department of Defense. He has been the recipient of the Hyundai Hope on Wheels Hope Award, St. Baldrick’s Scholar Award, and the American Brain Tumor Association Discovery Award.

Dr. Sayour is board-certified in general pediatrics and pediatric hematology-oncology.

He has presented his work at several national meetings and is a member of the Children’s Oncology Group, Society of Neuro-Oncology and the American Society of Pediatric Hematology-Oncology.

Certifications:
  • Hematology-Oncology
    American Board of Pediatrics
  • Pediatrics
    American Board of Pediatrics
Specialties:
  • Neurosurgery
  • Pediatrics
Subspecialties:
  • Pediatric Hematology-Oncology
Clinical Interests:
  • Brain tumor – children
Research Summary:
Research Interests:
  • Immunotherapeutics for CNS malignancies
  • Pediatric brain tumors
Publications:
Grants:
  • Dec 2025 ACTIVE
    Unlocking CAR T cells Activity Against Metastatic Osteosarcoma Using Systemic RNA Vaccination
    HYUNDAI HOPE ON WHEELS · Other
  • Nov 2025 ACTIVE
    Enhancing Immunotherapy Efficacy in NSCLC through Tumor Antigen-Targeted RNA Nanoparticles
    FL DEPT OF HLTH BIOMED RES PGM/J&E KING · Co-Investigator
  • Sep 2025 ACTIVE
    Developing a Functional Predictive Biomarker for an mRNA-LP Cancer Vaccine Clinical Trial Using Each Patients Own Living Tumor Tissue
    NATL PEDIATRIC CANCER FOU · Principal Investigator
  • Sep 2025 ACTIVE
    Overcoming metastatic spread of osteosarcoma with RNA loaded nanoparticles
    NATL INST OF HLTH NCI · Principal Investigator
  • Jun 2025 ACTIVE
    The Florida Summer Training in Research and Oncology for the Next Generation of Researchers (STRONGER) Program
    NATL INST OF HLTH NCI · Faculty
  • May 2025 ACTIVE
    NOVEL CORRELATIVE ASSESSMENTS OF PERFORMANCE OF QUAD-TARGETING CAR T CELLS FOR CHILDREN AND YOUNG ADULTS WITH DIPG OR DMG
    DIPG DMG RESEARCH FUNDING ALLIANCE · Principal Investigator
  • May 2025 ACTIVE
    ReSubmission R01 Role of bacterial-derived small molecules in immunotherapy
    NATL INST OF HLTH NCI · Co-Investigator
  • Feb 2025 – Jan 2026
    Multilamellar RNA aggregates targeting diffuse midline glioma, including diffuse intrinsic pontine glioma
    CHILDRENS NATIONAL RESEARCH INSTITUTE · Principal Investigator
  • Oct 2024 ACTIVE
    Nivolumab and DAY101 for the treatment of newly diagnosed or recurrent craniopharyngioma in children and young adults
    UNIV OF CALIFORNIA SAN FRANCISCO · Principal Investigator
  • Sep 2024 ACTIVE
    Targeted RNA-Lipid Particle Aggregates in Pediatric Oncology: A Novel Therapeutic Strategy for Fibrolamellar Hepatocellular Carcinoma
    HYUNDAI HOPE ON WHEELS · Other
  • Sep 2024 – Sep 2024
    Task Order Number: 15349sc PNOC COMP
    UNIV OF CALIFORNIA SAN FRANCISCO · Principal Investigator
  • Sep 2024 ACTIVE
    Task Order Number: 15349sc PNOC COMP
    UNIV OF CALIFORNIA SAN FRANCISCO · Principal Investigator
  • Jul 2024 ACTIVE
    Phase 2 Trial of G207 + 5 Gy Radiation for Children with High-Grade Gliomas
    ST JUDE CHILDRENS RESEARCH HOSPITAL · Principal Investigator
  • Jul 2024 ACTIVE
    HLX drives the G3 MB growth and is a potential candidate drug target to treat G3 MBs
    JOHNS HOPKINS ALL CHILDRENS HOSPITAL · Principal Investigator
  • Jul 2024 ACTIVE
    Combining Epigenetic Therapy and Immunotherapy to Target Group 4 Medulloblastoma
    CHILDRENS RESEARCH INSTITUTE · Co-Investigator
  • May 2024 – Jul 2024
    Pediatric Oncology Student Training (POST) Program
    ALEXS LEMONADE STAND FOUNDATION · Principal Investigator
  • May 2024 ACTIVE
    Combining Epigenetic Therapy and Immunotherapy to Target Group 4 Medulloblastoma
    CHILDRENS RESEARCH INSTITUTE · Project Manager
  • May 2024 ACTIVE
    B7-H3-CAR iNKT Therapy for High Risk Medulloblastoma
    NATL PEDIATRIC CANCER FOU · Principal Investigator
  • Apr 2024 ACTIVE
    Targeting Glycogen Metabolism in Ewing's Sarcoma: Diagnostic, Prognostic, and Therapeutic Applications
    NATL INST OF HLTH NCI · Co-Investigator
  • Feb 2024 – Jan 2026
    RNA-nanoparticle (RNA-NP) vaccine for ARMS
    KATE AMATO FOUNDATION · Other
  • Jul 2023 – Jun 2024
    Studying the impact of RNA-nanoparticle vaccine on immune response to DIPG/DMG
    DIPG DMG RESEARCH FUNDING ALLIANCE · Other
  • Jul 2023 – Feb 2025
    Nanoparticle vaccines targeting metastatic osteosarcoma
    OSTEOSARCOMA INSTITUTE · Principal Investigator
  • Jul 2023 ACTIVE
    Diabetes-Docs: Physician-Scientist Career Development Program (DiabDocs)
    STANFORD UNIV · Other
  • Jul 2023 ACTIVE
    The Sunshine Project: Development of RNA Loaded Nanoparticles for Children with Refractory Solid Tumors
    H LEE MOFFITT CANCER CTR & RES INST · Principal Investigator
  • Jun 2023 ACTIVE
    UF Health Cancer Center Support Grant
    NATL INST OF HLTH NCI · Other
  • Feb 2023 ACTIVE
    An RNA-based nanoparticle vaccine approach for the prevention and treatment of C9orf72 ALS/FTD
    US ARMY MED RES ACQUISITION · Co-Project Director/Principal Investigator
  • Jan 2023 ACTIVE
    Phase I trial of multilamellar mRNA lipid particles for recurrent pediatric high-grade glioma
    ALEXS LEMONADE STAND FOUNDATION · Principal Investigator
  • Jan 2023 – Dec 2024
    Investigating the Immune Response to RNA-lipid Nanoparticle Vaccines in a Clinical Trial for Canines with Osteosarcoma
    PEDIATRIC CANCER RESEARCH FOU · Other
  • Dec 2022 – Jun 2025
    Establishment of 3D metastatic osteosarcoma organoid model to accelerate combination immunotherapy
    HYUNDAI HOPE ON WHEELS · Other
  • Nov 2022 – Apr 2024
    Determining and enhancing stability of RNA-nanoparticle vaccines for a multi-center NPCF clinical trial for patients with metastatic osteosarcoma
    NATL PEDIATRIC CANCER FOU · Other
  • Sep 2022 – Aug 2025
    Overcoming metastatic spread of osteosarcoma with RNA loaded nanoparticles
    NATL INST OF HLTH NCI · Principal Investigator
  • Aug 2022 ACTIVE
    A phase I study of RNA-lipid particle vaccines for newly-diagnosed glioblastoma, IND19304 05/21/2020
    US FOOD AND DRUG ADMN · Principal Investigator
  • Jul 2022 ACTIVE
    Leveraging RNA-lipid nanoparticle vaccines to induce immune response in metastatic pulmonary osteosarcoma
    THE V FOU FOR CANCER RESEARCH · Co-Investigator
  • May 2022 – May 2025
    Patterns of resistance to initial therapeutic treatment in Ewings Sarcoma
    Hematology/Oncology Pharmacy Asso Fou · Co-Investigator
  • Apr 2022 – Mar 2025
    Unlocking CAR T cell efficacy against osteosarcoma using adjuvant RNA vaccine
    FL DEPT OF HLTH LIVE LIKE BELLA · Other
  • Apr 2022 ACTIVE
    Novel RNA-Nanoparticle vaccine for treatment of early melanoma recurrence following adjuvant anti-PD-1 antibody therapy
    FL DEPT OF HLTH BIOMED RES PGM/J&E KING · Co-Project Director/Principal Investigator
  • Feb 2022 – Feb 2022
    A Phase I Trial Evaluating the Combination of Trametinib and Everolimus in Pediatric and Young Adult Patients with Recurrent Low Grade Gliomas
    UNIV OF CALIFORNIA SAN FRANCISCO · Principal Investigator
  • Feb 2022 ACTIVE
    A Phase I Trial Evaluating the Combination of Trametinib and Everolimus in Pediatric and Young Adult Patients with Recurrent Low Grade Gliomas
    UNIV OF CALIFORNIA SAN FRANCISCO · Principal Investigator
  • Jan 2022 – Jun 2023
    Development of RNA Loaded Nanoparticles for Children with Refractory Solid Tumors
    H LEE MOFFITT CANCER CTR & RES INST · Principal Investigator
  • Jan 2022 – Dec 2024
    Development of RNA Loaded Nanoparticles for Children with Refractory Solid Tumors
    NATL PEDIATRIC CANCER FOU · Principal Investigator
  • Oct 2021 – Apr 2023
    Pan-Neoantigen Targeted RNA-nanoparticles for Recurrent Pulmonary Osteosarcoma
    CHANCE FOR LIFE FOUNDATION · Principal Investigator
  • Sep 2021 – Aug 2024
    Immune correlate studies in pediatric high-grade glioma patients receiving RNA nanoparticle vaccines
    TEAM JACK FOUNDATION · Principal Investigator
  • Jul 2021 – Aug 2022
    Immunotherapeutic targeting of osteosarcoma with nanoparticle vaccines
    Rally Foundation · Principal Investigator
  • Apr 2021 ACTIVE
    Proteolysis-targeting chimera against BCL-XL inhibits breast cancer metastasis
    NATL INST OF HLTH NCI · Co-Investigator
  • Feb 2021 – Jan 2026
    Overcoming the blood-brain barrier with nanoparticle vaccines against gliomas
    NATL INST OF HLTH NCI · Principal Investigator
  • Feb 2021 ACTIVE
    PNOC 021: A Phase I Trial Evaluating the Combination of Trametinib and Everolimus in Pediatric and Young Adult Patients with Recurrent Low Grade Gliomas
    UNIV OF CALIFORNIA SAN FRANCISCO · Principal Investigator
  • Jan 2021 – Jan 2026
    A Randomized, Double-Blinded, Pilot Trial of Neoadjuvant Checkpoint Inhibition followed by Combination Adjuvant Checkpoint Inhibition in Children and Young Adults with Recurrent or Progressive High Grade Glioma (HGG)
    UNIV OF CALIFORNIA SAN FRANCISCO · Principal Investigator
  • Jul 2020 ACTIVE
    Pediatric Brain Tumor ConsortiumA multi-institutional consortium devoted to novel phase I and II clinical evaluations of experimental treatment approaches for pediatric CNS tumors
    ST JUDE CHILDRENS RESEARCH HOSPITAL · Principal Investigator
  • Jul 2020 – Jul 2021
    Remodeling Host Immunity in Oral Cancer with Personalized RNA Nanoparticle Vaccines
    NATL INST OF HLTH NIDCR · Other
  • Jul 2020 – Jun 2024
    Translating Personalized RNA Nanoparticle Vaccines for Oral Cancer
    OCALA ROYAL DAMES FOR CANCER RESEARCH · Principal Investigator
  • Jul 2020 – Aug 2025
    RNA-nanoparticle vaccines for pediatric high-grade glioma
    CURESEARCH FOR CHILDRENS CANCER · Principal Investigator
  • Jun 2020 – Nov 2023
    Novel immunologic therapy of soft tissue sarcoma
    FL DEPT OF HLTH LIVE LIKE BELLA · Co-Investigator
  • Jun 2020 – May 2023
    Lipidnanoparticle vaccines targeting metastatic lung cancer from osteosarcoma
    FL DEPT OF HLTH BANKHEAD-COLEY CANCER RE · Principal Investigator
  • Jun 2020 – Nov 2023
    Multi-center phase I study evaluating lipid-nanoparticle vaccines against pediatric high-grade glioma
    FL DEPT OF HLTH LIVE LIKE BELLA · Principal Investigator
  • Jun 2020 ACTIVE
    A Phase I/II and Target Validation Study of TAK-580 (MLN2480) for Children with Low-Grade Gliomas and Other RAS/RAF/MEK/ERK Pathway Activated Tumors
    DANA FARBER CANCER INST · Principal Investigator
  • May 2020 – Jan 2022
    A Phase 1 Study of Modified Measles Virus (MV-NIS) for the Treatment of Children and Young Adults with Recurrent Medulloblastoma or Recurrent Atypical Teratoid Rhabdoid Tumors (ATRT)
    UNIV OF CALIFORNIA SAN FRANCISCO · Principal Investigator
  • Dec 2019 – Jun 2022
    Nanoparticles targeting genetic translocations in pediatric brain tumors
    HYUNDAI HOPE ON WHEELS · Principal Investigator
  • Sep 2019 – Oct 2023
    A Safety and Pharmacokinetic Study of Single Agent REGN2810 in Pediatric Patients with Relapsed or Refractory Solid or Central Nervous System (CNS) Tumors and a Safety and Efficacy Trial of REGN2810 in Combination with Radiotherapy in Pediatric Patients with Newly Diagnosed Diffuse Intrinsic Pontine Glioma, Newly Diagnosed High-Grade Glioma, or Recurrent High-Grade Glioma
    ICON CLINICAL RESEARCH INC · Principal Investigator
  • Aug 2019 ACTIVE
    A Target Validation Study of Fimepinostat in Children and Young Adults with Newly Diagnosed Diffuse Intristic Pontine Glioma (DIPG), Recurrent Medulloblastoma, or Recurrent High-Grade Glioma (HGG)
    UNIV OF CALIFORNIA SAN FRANCISCO · Principal Investigator
  • Jul 2019 – Dec 2020
    Re-programming immunity to target mutations across medulloblastoma subtypes
    ST BALDRICKS FOUNDATION · Principal Investigator
  • Jul 2019 – Sep 2021
    Immunotherapeutic targeting of osteosarcoma with nanoparticle vaccines
    Rally Foundation · Principal Investigator
  • Mar 2019 – Aug 2024
    CTOA
    UNIV OF FLORIDA · Principal Investigator
  • Jan 2019 – Dec 2023
    Development of personalized RNA loaded nano particles for children with refractory solid tumors.
    UF FOUNDATION · Principal Investigator
  • Dec 2018 – Mar 2022
    A Pilot Trial Testing the Clinical Benefit of Using Molecular Profiling to Determine an Individualized Treatment Plan in Children and Young Adults with High Grade Glioma (Excluding Diffuse Intrinsic Pontine Glioma)
    UNIV OF CALIFORNIA SAN FRANCISCO · Principal Investigator
  • Jul 2018 – Jun 2020
    Host Immunomodulatory RNA-nanoparticles targeting malignant canine glioma
    UF FOUNDATION · Principal Investigator
  • Jul 2018 – Dec 2021
    Legacy Foundation
    UF FOUNDATION · Project Manager
  • Jul 2018 – Jun 2025
    Florida Center for Brain Tumor Research
    FL DEPT OF HLTH · Project Manager
  • Mar 2018 ACTIVE
    Safety, Phase 0, and Pilot Efficacy Study of Vemurafenib, an Oral Inhibitor of BRAFV600E, in Children and Young Adults with Recurrent/Refractory BRAFV600E – or BRAF Ins T mutant Brain Tumors
    UNIV OF CALIFORNIA SAN FRANCISCO · Principal Investigator
  • Jan 2018 – Jun 2020
    Development of Personalized RNA Loaded Nanoparticles for Children with Refractory Solid Tumors
    H LEE MOFFITT CANCER CTR & RES INST · Principal Investigator
  • Dec 2017 – Nov 2024
    Phase II Study of Everolimus for Recurrent or Progressive Low-grade Gliomas in Children
    UNIV OF CALIFORNIA SAN FRANCISCO · Principal Investigator
  • Nov 2017 – Nov 2025
    Adoptive Cellular Therapy following Dose-Intensified Temozolomide in Newly-diagnosed Pediatric High-grade Gliomas (Phase I)
    H LEE MOFFITT CANCER CTR & RES INST · Principal Investigator
  • Sep 2017 – Sep 2020
    RNA-nanoparticles targeting H3.3 K27M epitopes in diffuse intrinsic pontine glioma
    US ARMY MED RES ACQUISITION · Principal Investigator
  • Sep 2017 – Jun 2021
    UFHCC Triple Negative Breast Cancer (TNBC) Pilot Project Program
    MULTIPLE SPONSORS · Project Manager
  • Jul 2017 – Dec 2019
    Immunotherapeutic targeting of osteosarcoma neoantigens
    Rally Foundation · Principal Investigator
  • May 2017 – Feb 2023
    Phase Ib /II Clinical Trial of Nivolumab Monotherapy and Nivolumab in Combination with Ipilimumab in Pediatric Subjects with High Grade Primary CNS Malignancies
    BRISTOL MYERS SQUIBB CO · Principal Investigator
  • Apr 2017 – Jun 2021
    OoR CTSI Institutional Matching Support
    UF DIV OF SPONSORED RES MATCHING FUNDS · Project Manager
  • Nov 2016 ACTIVE
    Newly Diagnosed Children (Less than 10 Years Old) With Medulloblastoma and Other Central Nervous System Primitive Neuro-Ectodermal Tumors: Clinical and Molecular Risk-Tailored Intensive and Compressed Induction Chemotherapy Followed by Consolidation with Either Single-Cycle (Low Risk Patients) or Randomization (High Risk Patients) to either Single-Cycle or to Three Tandem Cycles of Marrow-Ablative Chemotherapy with Autologous Hematopoietic Progenitor Cell Rescue
    NATIONWIDE CHILDRENS HOSPITAL · Principal Investigator
  • Sep 2016 ACTIVE
    A Clinical and Molecular Risk-directed Therapy for Newly Diagnosed Medulloblastoma
    ST JUDE CHILDRENS RESEARCH HOSPITAL · Principal Investigator
  • Sep 2016 – Aug 2021
    Harnessing the Immunologic Capacity of RNA-nanoparticle Vaccines Targeting Glioblastoma
    NATL INST OF HLTH NCI · Principal Investigator
  • Jul 2016 – Jun 2020
    RNA nanoparticle vaccines targeting malignant canine gliomas
    OCALA ROYAL DAMES FOR CANCER RESEARCH · Principal Investigator
  • Jul 2016 – Jun 2020
    Re-programming immunity to target mutations across medulloblastoma subtypes
    ST BALDRICKS FOUNDATION · Principal Investigator
  • Apr 2016 – Mar 2018
    Re-programming osteosarcoma immune evasion with CAR modified T cells
    UF FOUNDATION · Principal Investigator
  • Jul 2015 – Jun 2024
    Florida Academic Cancer Center Alliance Seed Grants
    UF HEALTH SHANDS HOSPITAL · Project Manager
  • Dec 2014 – Jun 2017
    Efficacious, Translational and Personalized RNA-nanoparticle Vaccines Targeting Pediatric Brain Tumors
    HYUNDAI HOPE ON WHEELS · Principal Investigator
  • Jul 2013 – Jun 2021
    Florida Center for Brain Tumor Research – Statewide Brain Tumor Registry Program at the McKnight Brain Institute
    FL DEPT OF HLTH · Project Manager
Patents:
  • Published May 2023
    Materials and Methods for Sensitizing Tumors to Immune Response
    #2023-0414721-A1
  • Published September 2022
    Multilamellar RNA Nanoparticle Vaccine Against SARS-COV-2
    #US-2023-0226169-A1
  • Published August 2022
    Multilamellar RNA Nanoparticles and Methods of Sensitizing Tumors to Treatment with Immune Checkpoint Inhibitors
    #US-2023-0346700-A1
  • Published August 2022
    RNA-Loaded Nanoparticles and Use Thereof for the Treatment of Cancer
    #US-2023-0096704-A1
  • Published October 2021
    Compositions for Treatment of Diffuse Intrinsic Pontine Glioma
    #US-2022-0218808-A1
  • Published February 2021
    Methods of Sensitizing Tumors to Treatment with Immune Checkpoint Inhibitors
    #US-2021-0170005-A1
  • Published November 2020
    Magnetic Liposomes and Related Treatment and Imaging Methods
    #US-2021-0077399-A1
  • Published February 2020
    A Monophosphoryl Lipid A Liposome Based Cancer Vaccine
    #US-2022-0133872-A1
Education:
  • 2013
    Fellowship – Pediatric Hematology/Oncology
    Duke University Medical Center
  • 2012-2015
    Ph.D.
    Duke University
  • 2010
    Residency – Pediatrics
    Long Island Jewish Medical Center
  • 2007
    M.D.
    University at Buffalo
  • 2003
    B.S.
    Fordham University
Ciaran Seath

Ciaran Seath

AST PROF
Department: SR-CHEM-SEATH LAB
Phone: (561) 228-2672
Physical Address:

Ciaran completed his PhD in chemistry from the University of Strathclyde in 2017 under the supervision of Dr Allan Watson studying chemoselective transition-metal catalysis. During this time he also spent time in the labs of Prof. Tom Snaddon at Indiana University working on total synthesis of complex natural products, and with Prof. Glenn Burley on the development of novel click chemistries for nucleoside bioconjugation.

He then moved to Emory University as a postdoctoral researcher working in the laboratory of Professor Nathan Jui developing novel reductive photoredox methodologies. During this time, Ciaran worked on new synthetic methods to access alkylated heterocycles and complex fluorinated scaffolds.

In 2019, Ciaran moved to Princeton University to work with Professors David MacMillan and Tom Muir where he explored new photocatalytic methods for proximity labelling to investigate critical aspects of cancer biology.

Currently, Ciaran is an Assistant Professor of Chemistry at Scripps-UF in Jupiter, Florida.

Research Summary:

A critical component of the development of new medicines is the identification and validation of new protein targets. Our laboratory is focused on using state-of the-art methods in chemical biology to discover new therapeutically relevant protein-biomolecule interactions that contribute to disease.

Research Interests:
  • Biochemistry and Cell Biology
  • Clinical and translational science
  • Pediatric brain tumors
Publications:
Grants:
  • Mar 2025 ACTIVE
    Nanoscale proximity labeling for chromatin chemical biology
    PFIZER INC · Principal Investigator
  • Jun 2024 – May 2025
    Vanquish Neo UHPLC for The Wertheim UF Scripps Institute
    NATL INST OF HLTH OD · Other
  • Jan 2024 ACTIVE
    Photocatalytic Generation of Siloxycarbenes for Asymmetric Organocatalysis
    AMER CHEMICAL SOC PETRO RES · Principal Investigator
  • Jul 2023 ACTIVE
    Photoproximity labeling as a tool for epigenetic drug discovery
    NATL INST OF HLTH NIGMS · Principal Investigator
  • May 2023 ACTIVE
    Mechanisms of estrogen receptor ligand signaling
    NATL INST OF HLTH NCI · Co-Investigator
Maurice S Swanson

Maurice S Swanson

Professor and Associate Program Director
Department: Molecular Genetics & Microbiology
Phone: (352) 273-8076
Mailing Address:
Physical Address:

Fun Fact: I love to snow ski and play ice hockey.

Our research group focuses on how RNA processing is controlled during mammalian development and how this regulation is disrupted in neurological and neuromuscular diseases. More than half of the human genome is comprised of repetitive elements, including interspersed and tandem repeats, and more than 60 hereditary diseases are caused by the expansion of short tandem repeats (STRs). We investigate the functions of these genomic elements using a wide array of biochemical (e.g., transcriptomics, proteomics) and genetic (e.g., gene knockout and knockin models) strategies. Additionally, we study how RNA binding proteins (RBPs) interact with various RNA structures using global analysis of RBP binding sites, the functions of nuclear RNA foci (biomolecular condensates) generated by pathogenic STR expansions, and how RNA-based mechanisms promote mammalian tissue regeneration. Finally, we are developing novel cell and mouse models to test therapeutic strategies designed to block the toxicity of repetitive element mutations.

Research Interests:
  • Aging
  • Developmental regulation of RNA processing
  • Genetics of Neurological diseases
  • Mammalian genome repetitive elements
  • Mechanisms of Regeneration
  • Myotonic Dystrophy
  • Neurological disorders
  • RNA binding proteins
Publications:
Grants:
  • Aug 2024 ACTIVE
    Senator Paul D. Wellstone Muscular Dystrophy Specialized Research Center
    NATL INST OF HLTH NINDS · Principal Investigator
  • Jan 2024 ACTIVE
    The molecular basis of 7SK RNA methylation in non-small cell lung cancer
    NATL INST OF HLTH NCI · Co-Investigator
  • Sep 2023 – Jul 2024
    Mouse Dmpk CTG expansion knock in models for myotonic dystrophy (MDA 1070134)
    MUSCULAR DYSTROPHY ASSO · Other
  • Sep 2023 ACTIVE
    Understanding and targeting common mechanisms of myotonic dystrophy types 1 and 2 using novel repeat expansion mouse models
    US ARMY MED RES ACQUISITION · Principal Investigator
  • Jan 2023 – Jun 2025
    Regenerative failure in myotonic dystrophy: pathomechanisms and insights from a novel model of improved regeneration
    MYOTONIC DYSTROPHY FOU · Other
  • Jan 2023 ACTIVE
    Therapeutic strategies for microsatellite expansion diseases using RNA-targeting
    UNIV OF CALIFORNIA SAN DIEGO · Principal Investigator
  • Aug 2021 – Aug 2022
    Mouse humanized models for myotonic dystrophy type 1 for therapy development
    KATE THERAPEUTICS · Principal Investigator
  • Jul 2021 – Sep 2023
    Brain Choroid Plexus Dysregulation and Cerebral Atrophy in DM1
    MYOTONIC DYSTROPHY FOU · Other
  • Jan 2020 – Jan 2023
    Provenance Initiatives Fund at the Center for NeuroGenetics
    UF FOUNDATION · Co-Investigator
  • Jul 2019 – Jun 2025
    RNA Processing-Mediated Mechanisms of CNS Dysfunction in Myotonic Dystrophy
    NATL INST OF HLTH NINDS · Faculty
  • Jun 2019 – Aug 2019
    International Myotonic Dystrophy Consortium Meeting IDMC-12
    MUSCULAR DYSTROPHY ASSO · Principal Investigator
  • Sep 2018 – Aug 2023
    Senator Paul D. Wellstone Muscular dystrophy Speciallzed Research Center (Project 2 Disease Progress and Biomarkers)
    UNIV OF ROCHESTER · Principal Investigator
  • Sep 2018 – Aug 2023
    Senator Paul D. Wellstone Muscular Dystrophy Specialized Research Center (Project 3 Preclinical Model and Therapies for DM2).
    UNIV OF ROCHESTER · Principal Investigator
  • Sep 2018 – Aug 2023
    Senator Paul D. Wellstone Muscular Dystrophy Specialized Research Center (Administrative Core)
    UNIV OF ROCHESTER · Principal Investigator
  • Sep 2018 – Aug 2024
    Senator Paul D. Wellstone Muscular Dystrophy Specialized Research Center
    UNIV OF ROCHESTER · Principal Investigator
  • Feb 2018 – Jan 2021
    Pre-mRNA Misprocessing in Myotonic Dystrophy and Amyotrophic Lateral Sclerosis
    MUSCULAR DYSTROPHY ASSO · Other
  • Jan 2018 – Dec 2019
    Congenital myotonic dystrophy:pathomechanism and therapeutic development
    WYCK FOUNDATION · Other
  • Sep 2017 – May 2022
    Therapeutic strategies for microsatellite expansion diseases using RNA-targeting CRISPR/Cas
    UNIV OF CALIFORNIA SAN DIEGO · Principal Investigator
  • Aug 2017 – Apr 2022
    Determining the factors that control dose-dependent splicing regulation by a master regulator
    NATL INST OF HLTH NIGMS · Principal Investigator
  • Feb 2017 – Jan 2020
    Novel Mouse Knockin Models for Myotonic Dystrophy
    MUSCULAR DYSTROPHY ASSO · Principal Investigator
  • Aug 2016 – Jun 2022
    Molecular Characterization of ALS/FTD in a novel C9orf72 BAC mouse model.
    NATL INST OF HLTH NINDS · Principal Investigator
  • Feb 2016 – Jan 2018
    Myotonic Dystrophy Type 2: Mouse Models, pathomechanism and therapy
    WYCK FOUNDATION · Principal Investigator
  • Jan 2015 – Jan 2018
    MBI Research Program MGOLD2
    UF FOUNDATION · Principal Investigator
  • Sep 2013 – Jun 2019
    Myotonic Dystrophy: Molecular Pathophysiology and CNS Effects
    NATL INST OF HLTH NINDS · Project Manager
  • Aug 2013 – Jul 2016
    Circadian Clock Dysregulation in Myotonic Dystrophy
    MUSCULAR DYSTROPHY ASSO · Principal Investigator
  • Jul 2013 – Jun 2018
    Research Program MGOLD project 2
    UF FOUNDATION · Principal Investigator
  • Aug 2010 – Jun 2016
    RNA Dominance in Human Disease
    NATL INST OF HLTH · Principal Investigator
  • Jul 2006 – Sep 2024
    BRAIN AND SPINAL CORD INJURY RESEARCH
    FL DEPT OF HLTH · Project Manager
Patents:
  • Issued June 2016
    Compositions and Methods Related to Protein Displacement Therapy for Myotonic Distrophy (CON)
    #9,371,527
  • Issued June 2011
    Methods and Compositions for Treatment of Myotonia
    #7,964,570
  • Published March 2010
    Compositions and Methods Related to Protein Displacement Therapy for Myotonic Distrophy
    #US-2010-0190689-A1
Education:
  • 1979
    PhD
    University of California Berkeley
  • 1976
    MA
    University of California Santa Barbara
Scott Tibbetts

Scott Tibbetts

Professor
Department: Molecular Genetics & Microbiology
Phone: (352) 273-5628
Mailing Address:
Physical Address:

Fun Fact: I love traveling, travel photography, and learning about other cultures through experiences, conversations, and food; being able to be a part of a multi-cultural team is one of my favorite aspects of academic science. Our lab studies gammaherpesviruses – viruses such as Epstein-Barr virus (EBV) and Kaposi’s sarcoma herpesvirus (KSHV, HHV-8), which are associated with multiple types of human malignancies, including lymphomas, carcinomas, and sarcoma. Our research harnesses the power of the closely related murine gammaherpesvirus (MHV68) in vivo system to gain insight into the mechanisms by which viral noncoding RNAs contribute to latency and tumorigenesis, and to define the molecular and pathogenic strategies that these viruses use to evade the immune system and manipulate B cell biology in order to establish lifelong latent infections. The long-term goals of these studies are to better understand the complex interplay between these viruses and the host immune response, and define how alterations in this relationship can result in the development of lymphoproliferative diseases and lymphoma, and elucidate new targets for prevention of gammaherpesvirus malignancies.

Publications:
Grants:
  • Jun 2022 ACTIVE
    Noncoding RNAs in gamma-Herpesvirus Biology and AIDS Malignancies
    NATL INST OF HLTH NCI · Co-Investigator
  • Aug 2021 ACTIVE
    Gammaherpesvirus miRNA suppression of EWSR1 in GC B cell infection and lymphomagenesis
    NATL INST OF HLTH NCI · Principal Investigator
  • Jul 2018 – Aug 2019
    The Role of a Gammaherpesvirus Noncoding RNA in Dissemination, Chronic Infection and Virus-Induced Vasculitis
    AMER HEART ASSOCIATION · Other
  • Feb 2017 – Jan 2022
    Noncoding RNAs in gamma-Herpesvirus Biology and AIDS Malignancies
    NATL INST OF HLTH NCI · Faculty
  • Feb 2015 – Oct 2023
    UF Health Cancer Center Pilot Project Grants funded through the Florida Consortium of National Cancer Institute Centers Program
    UF HEALTH SHANDS HOSPITAL · Project Manager
  • Jan 2015 – Dec 2020
    Role of MHV68 miRNAs in latency and pathogenesis
    NATL INST OF HLTH NIAID · Principal Investigator
  • Jan 2015 – May 2024
    UF Health Cancer Center Bridge Seed Grant funded through the Florida Consortium of National Cancer Institute Centers Program
    UF HEALTH SHANDS HOSPITAL · Project Manager
  • Mar 2006 – May 2023
    Davis Cancer Researach Equipment
    UF FOUNDATION · Project Manager