Jessica Childs-Disney
- Mailing Address:
- 2025 RSC chemical biology
- 2025 Journal of medicinal chemistry
- 2025 ACS chemical biology
- 2024 bioRxiv : the preprint server for biology
- 2024 Journal of the American Chemical Society
- 2024 British journal of pharmacology
- 2023 Expert opinion on drug discovery
- 2023 Journal of the American Chemical Society
- 2023 Journal of medicinal chemistry
- 2023 Nature
- 2023 Nucleic acids research
- 2023 ACS central science
- 2023 Methods in enzymology
- 2023 Scientific reports
- 2023 ACS chemical biology
- 2022 Proceedings of the National Academy of Sciences of the United States of America
- 2022 Journal of the American Chemical Society
- 2022 Nature reviews. Drug discovery
- 2022 Proceedings of the National Academy of Sciences of the United States of America
- 2021 Current opinion in chemical biology
- 2021 Journal of medicinal chemistry
- 2021 ACS chemical biology
- 2021 Journal of the American Chemical Society
- 2021 Science translational medicine
- 2020 Proceedings of the National Academy of Sciences of the United States of America
- 2020 Proceedings of the National Academy of Sciences of the United States of America
- 2020 Journal of the American Chemical Society
- 2020 Journal of the American Chemical Society
- 2020 Current opinion in chemical biology
- 2020 ACS chemical biology
- 2020 Journal of medicinal chemistry
- 2020 Chemical Society reviews
- 2020 Chemical Society reviews
- 2019 Cell chemical biology
- 2019 Journal of the American Chemical Society
- 2019 Methods in enzymology
- 2019 PloS one
- 2019 PloS one
- 2018 Chemical reviews
- 2018 Cell chemical biology
- 2018 Chem
- 2018 Cold Spring Harbor perspectives in biology
- 2017 Methods in molecular biology (Clifton, N.J.)
- 2017 Journal of the American Chemical Society
- 2017 ACS central science
- 2016 ACS chemical biology
- 2016 BMC bioinformatics
- 2016 ACS chemical biology
- 2016 Annual review of pharmacology and toxicology
- 2014 ACS chemical biology
- 2014 Organic & biomolecular chemistry
- 2014 ACS chemical biology
- 2013 Nature communications
- 2012 Journal of the American Chemical Society
- 2012 ACS chemical biology
- 2012 ACS chemical biology
- 2012 ACS chemical biology
- 2012 ACS chemical biology
- 2011 Chembiochem : a European journal of chemical biology
- 2010 Chembiochem : a European journal of chemical biology
- 2009 Journal of the American Chemical Society
- 2009 Nucleic acids research
- 2009 Journal of the American Chemical Society
- 2008 RNA (New York, N.Y.)
- 2008 Journal of the American Chemical Society
- 2008 Biochemistry
- 2007 Chembiochem : a European journal of chemical biology
- 2007 Chemistry & biology
- 2007 ACS chemical biology
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Oct 2025
ACTIVE
Novel mRNA degraders to target hypoxic tumor microenvironmentFL DEPT OF HLTH BANKHEAD-COLEY CANCER RE · Co-Project Director/Principal Investigator
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Feb 2025
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Jan 2026
ASD gene therapy: Large-scale evaluation of small-molecule targeting of RNA for ASD therapeuticsICAHN SCHOOL OF MEDICINE AT MOUNT SINAI · Principal Investigator
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Jun 2024
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Dec 2025
Developing Artificial Intelligence and Machine Learning Approaches to Identify and Optimize Small Molecule Medicines Targeting Oncogenic RNAsFL DEPT OF HLTH · Principal Investigator
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Mar 2024
ACTIVE
Design of small molecules that degrade the mRNA encoding a-synucleinSPARK NS · Co-Project Director/Principal Investigator
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Apr 2022
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Nov 2023
Advance Therapeutic Targets & Biomarkers for ALS FTDTARGET ALS FOUNDATION · Principal Investigator
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Apr 2022
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Feb 2024
Targeting RNAs Encoding Proteins Associated with Autism Spectrum DisordersICAHN SCHOOL OF MEDICINE AT MOUNT SINAI · Principal Investigator
Matthew Disney Ph.D.
- Mailing Address:
- Physical Address:
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Recipient2021 · The ACS 2022 Nobel Laureate Signature Award for Graduate Education in Chemistry
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Awarded2019 · The Raymond and Beverly Sackler International Prize in Chemistry
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The Barry Cohen Prize2018 · Medicinal Chemistry Section of the Israel Chemical Society and Teva Pharmaceutical Industries.
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Awarded2018 · BioFlorida's Weaver H. Gaines Entrepreneur of the Year
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Outstanding Mentor Award2017 · Scripps Florida
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Tetrahedron Young Investigator Award in Bioorganic and Medicinal Chemistry2016 · —
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Blavatnik Young Scientists Award Finalist2015-2017 · —
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NIH Director’s Pioneer Award2015 · National Institutes of Health
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David W. Robertson Award in Medicinal Chemistry2014 · American Chemical Society’s Division of Medicinal Chemistry
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Excellence Award in the field of Research in Science and Technology2013 · India-US Chamber of Commerce, Inc.
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Eli Lilly Award in Biological Chemistry2013 · American Chemical Society
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David Gin Award in Carbohydrate Chemistry2012 · American Chemical Society
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Dreyfus Teacher-Scholar Award2010-2015 · —
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Excellent Scholar, Young Investigator Award2010 · University at Buffalo
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Research Corporation Cottrell Scholar Award2008-2010 · —
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NYSTAR JD Watson Young Investigator Award2007-2009 · —
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Camille and Henry Dreyfus New Faculty Award2005-2010 · —
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Second Year Roche Foundation Postdoctoral Fellowship2004-2005 · Swiss Federal Institute of Technology
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Roche Foundation Postdoctoral Fellowship2003-2004 · Swiss Federal Institute of Technology
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Arnold Weissberger Memorial Fellow2001-2002 · University of Rochester
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Elon Huntington Hooker Memorial Fellow2000-2001 · University of Rochester
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Sherman-Clark Memorial Fellow1997-1999 · University of Rochester
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Eric A. Batista Award1997 · University of Maryland at College Park
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Howard Hughes Medical Institute Undergraduate Research Fellow1995-1997 · University of Maryland at College Park
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.
- 2025 RSC Chemical Biology
- 2025 Biochemistry
- 2025 Nucleic Acids Research
- 2025 Journal of Medicinal Chemistry
- 2025 ACS Chemical Biology
- 2025 bioRxiv : the preprint server for biology
- 2025 ACS Chemical Biology
- 2025 Journal of the American Chemical Society
- 2025 ACS Central Science
- 2025 Journal of the American Chemical Society
- 2025 American journal of respiratory cell and molecular biology
- 2025 Journal of the American Chemical Society
- 2025 Journal of the American Chemical Society
- 2025 Journal of the American Chemical Society
- 2024 Proceedings of the National Academy of Sciences
- 2024 Trends in Pharmacological Sciences
- 2024 bioRxiv : the preprint server for biology
- 2024 Journal of the American Chemical Society
- 2024 STAR protocols
- 2024 Bioorganic & medicinal chemistry letters
- 2024 British journal of pharmacology
- 2023 Expert opinion on drug discovery
- 2023 Journal of the American Chemical Society
- 2023 Nature
- 2023 Journal of Medicinal Chemistry
- 2023 ACS Central Science
- 2023 Israel Journal of Chemistry
- 2023 Methods in enzymology
- 2023 Neurology and Therapy
- 2023 ACS Chemical Biology
- 2023 ACS Chemical Biology
- 2022 ACS Chemical Biology
- 2022 Proceedings of the National Academy of Sciences
- 2022 Cell chemical biology
- 2022 Journal of the American Chemical Society
- 2022 Nature Reviews Drug Discovery
- 2022 Journal of the American Chemical Society
- 2022 Journal of the American Chemical Society
- 2022 Journal of the American Chemical Society
- 2022 Proceedings of the National Academy of Sciences
- 2021 Cell chemical biology
- 2021 ACS Medicinal Chemistry Letters
- 2021 Faculty Opinions – Post-Publication Peer Review of the Biomedical Literature
- 2021 Faculty Opinions – Post-Publication Peer Review of the Biomedical Literature
- 2021 ACS Chemical Biology
- 2021 Current Opinion in Chemical Biology
- 2021 NAR genomics and bioinformatics
- 2021 Cell
- 2021 Journal of Medicinal Chemistry
- 2021 Human molecular genetics
- 2021 Journal of the American Chemical Society
- 2021 Journal of the American Chemical Society
- 2021 Science Translational Medicine
- 2021 ACS Chemical Neuroscience
- 2021 ACS Chemical Biology
- 2020 Chemical Society Reviews
- 2020 Chemical Society Reviews
- 2020 Proceedings of the National Academy of Sciences
- 2020 ACS Chemical Biology
- 2020 Proceedings of the National Academy of Sciences
- 2020 Cell chemical biology
- 2020 ACS Chemical Biology
- 2020 Journal of Medicinal Chemistry
- 2020 Journal of the American Chemical Society
- 2020 bioRxiv : the preprint server for biology
- 2020 Cell reports
- 2020 Journal of the American Chemical Society
- 2020 Current Opinion in Chemical Biology
- 2020 ACS Chemical Biology
- 2020 ACS Chemical Biology
- 2020 Journal of Medicinal Chemistry
- 2020 ChemBioChem
- 2020 Nature Chemistry
- 2020 ACS Central Science
- 2020 SLAS Discovery
- 2020 ACS Chemical Biology
- 2020 Proceedings of the National Academy of Sciences
- 2019 Cell chemical biology
- 2019 Molecular neurodegeneration
- 2019 Journal of the American Chemical Society
- 2019 Journal of the American Chemical Society
- 2019 Annals of the New York Academy of Sciences
- 2019 Proceedings of the National Academy of Sciences of the United States of America
- 2019 Cell chemical biology
- 2019 Surveys in geophysics
- 2019 Methods in enzymology
- 2019 PloS one
- 2019 Journal of the Royal Society, Interface
- 2019 Bioorganic & medicinal chemistry letters
- 2019 PLOS ONE
- 2019 Cell chemical biology
- 2019 Faculty Opinions – Post-Publication Peer Review of the Biomedical Literature
- 2019 Faculty Opinions – Post-Publication Peer Review of the Biomedical Literature
- 2019 Faculty Opinions – Post-Publication Peer Review of the Biomedical Literature
- 2019 Faculty Opinions – Post-Publication Peer Review of the Biomedical Literature
- 2019 Faculty Opinions – Post-Publication Peer Review of the Biomedical Literature
- 2019 Faculty Opinions – Post-Publication Peer Review of the Biomedical Literature
- 2019 Faculty Opinions – Post-Publication Peer Review of the Biomedical Literature
- 2019 Drug discovery today
- 2018 Chemical Reviews
- 2018 Journal of the American Chemical Society
- 2018 ACS Combinatorial Science
- 2018 Bioorganic & medicinal chemistry letters
- 2018 Bioorganic & medicinal chemistry letters
- 2018 Cell chemical biology
- 2018 ACS Chemical Biology
- 2018 Bioorganic & medicinal chemistry
- 2018 Chem
- 2018 Cold Spring Harbor perspectives in biology
- 2018 The Journal of Physical Chemistry B
- 2017 Methods in molecular biology (Clifton, N.J.)
- 2017 Journal of the American Chemical Society
- 2017 Science translational medicine
- 2017 ACS Central Science
- 2017 Biochemistry
- 2017 Trends in biochemical sciences
- 2017 ChemBioChem
- 2016 ACS chemical biology
- 2016 BMC bioinformatics
- 2016 Proceedings of the National Academy of Sciences of the United States of America
- 2016 ACS chemical biology
- 2016 Nature Communications
- 2016 Bioorganic & medicinal chemistry letters
- 2016 Angewandte Chemie (International ed. in English)
- 2016 ACS chemical biology
- 2016 Bioorganic & medicinal chemistry
- 2016 Annual review of pharmacology and toxicology
- 2016 BMC structural biology
- 2016 Accounts of chemical research
- 2016 Nature Chemical Biology
- 2016 Bioorganic & medicinal chemistry letters
- 2015 Current opinion in chemical biology
- 2015 Journal of the American Chemical Society
- 2015 Neuron
- 2015 ACS chemical biology
- 2015 Journal of chemical theory and computation
- 2015 Bioorganic & medicinal chemistry letters
- 2015 ACS chemical biology
- 2014 ACS chemical biology
- 2014 Organic & biomolecular chemistry
- 2014 Chemical communications (Cambridge, England)
- 2014 ACS chemical biology
- 2014 Nature chemical biology
- 2014 Nucleic acids research
- 2014 Neuron
- 2014 Chembiochem : a European journal of chemical biology
- 2014 Neuron
- 2014 Angewandte Chemie (International ed. in English)
- 2013 Angewandte Chemie (International ed. in English)
- 2013 Cell reports
- 2013 Angewandte Chemie (International ed. in English)
- 2013 ACS chemical biology
- 2013 Bioorganic & medicinal chemistry
- 2013 Drug discovery today
- 2013 Nature communications
- 2012 ACS chemical biology
- 2012 Methods in molecular biology (Clifton, N.J.)
- 2012 Journal of the American Chemical Society
- 2012 ACS chemical biology
- 2012 ACS chemical biology
- 2012 ACS chemical biology
- 2012 ACS chemical biology
- 2012 ACS chemical biology
- 2011 Biochemistry
- 2011 Biochemistry
- 2011 Chembiochem : a European journal of chemical biology
- 2011 Chembiochem : a European journal of chemical biology
- 2011 Biochemistry
- 2010 Chembiochem : a European journal of chemical biology
- 2010 Chembiochem : a European journal of chemical biology
- 2009 ACS chemical biology
- 2009 Journal of the American Chemical Society
- 2009 Journal of the American Chemical Society
- 2008 RNA (New York, N.Y.)
- 2008 Journal of the American Chemical Society
- 2008 Biochemistry
- 2007 Chembiochem : a European journal of chemical biology
- 2007 Chemistry & biology
- 2007 ACS chemical biology
- 2005 Antimicrobial agents and chemotherapy
- 2004 Proceedings of the National Academy of Sciences of the United States of America
- 2001 Biochemistry
- 2001 Biochemistry
- 2000 Biochemistry
- 2000 Biochemistry
- 1999 Biochemistry
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Oct 2025
ACTIVE
Novel mRNA degraders to target hypoxic tumor microenvironmentFL DEPT OF HLTH BANKHEAD-COLEY CANCER RE · Co-Investigator
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Apr 2025
ACTIVE
Targeted degradation of RNAs by using small moleculesNATL INST OF HLTH NCI · Principal Investigator
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Dec 2024
ACTIVE
Small molecule degradation of tau mRNARAINWATER CHARITABLE FOUNDATION · Principal Investigator
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Jun 2024
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Dec 2025
Developing Artificial Intelligence and Machine Learning Approaches to Identify and Optimize Small Molecule Medicines Targeting Oncogenic RNAsFL DEPT OF HLTH · Co-Project Director/Principal Investigator
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Mar 2024
ACTIVE
Design of small molecules that degrade the mRNA encoding a-synucleinSPARK NS · Principal Investigator
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Dec 2023
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Nov 2024
Brain penetrant small molecules drugs that target tau pre-mRNA for degradationRAINWATER CHARITABLE FOUNDATION · Principal Investigator
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Oct 2023
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Oct 2025
Astellas Pharma Inc. bench fees for Dr. Ryuichi SekiokaASTELLAS PHARMA US · Principal Investigator
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Sep 2023
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Aug 2025
Graduate Research Fellowship program (GRFP)SCRIPPS RESEARCH INST · Principal Investigator
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Sep 2023
ACTIVE
Targeting the RNA Repeat Expansion that Causes Myotonic Dystrophy Type 2MUSCULAR DYSTROPHY ASSO · Principal Investigator
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Sep 2023
ACTIVE
Development of Clinical Candidates for the Treatment of Myotonic DystrophyUS ARMY MED RES ACQUISITION · Principal Investigator
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Apr 2023
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Jun 2025
CD33 Targeting Small Molecule Drug DiscoveryEISAI CO · Principal Investigator
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Sep 2022
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Aug 2023
Expanding Ribonuclease Targeting Chimeras (RIBOTACs) to Advance Targeted Degradation of Disease-Causing RNAsAMER CHEMICAL SOC · Other
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Sep 2022
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Aug 2025
Design and evaluation of drug-like small molecules targeting repeat expansion (MDA 963835)MUSCULAR DYSTROPHY ASSO · Other
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May 2022
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Apr 2025
Development of outpatient antiviral cocktails against SARS-CoV-2 and other potential pandemic RNA virusesSTANFORD UNIV · Principal Investigator
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May 2022
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Apr 2025
Center for Antiviral Medicines & Pandemic Preparedness (CAMPP) – PROJECT 4SCRIPPS RESEARCH INST · Principal Investigator
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Apr 2022
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Dec 2022
Small Molecule Targeting of DUX4 mRNA for treatment of Fascioscapulohumeral Dystrophy (FSHD)PFIZER INC · Principal Investigator
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Apr 2022
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Dec 2022
Developing Pre-Clinical Candidates Against miRNAs Implicated in Autoimmune DiseasesEMD SERONO RESEARCH & DEVELOPMENT INST · Principal Investigator
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Apr 2022
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Apr 2023
Pathophysiology of genetically defined dementia and neurodegeneration: Defining therapeutic targets and pathwaysNATL INST OF HLTH NINDS · Principal Investigator
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Apr 2022
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May 2023
RNA-Targeted Drug Discovery and Development for Parkinson's DiseaseRUTGERS STATE UNIV · Principal Investigator
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Apr 2022
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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
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Apr 2022
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Oct 2023
Targeting RNAs Associated with Tauopathies with Small MoleculesRAINWATER CHARITABLE FOUNDATION · Principal Investigator
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Apr 2022
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Oct 2023
Pre-clinical evaluation of small molecules that rescue aberrant Tau mRNA splicingRAINWATER CHARITABLE FOUNDATION · Principal Investigator
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Apr 2022
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Aug 2024
Small Molecules That Target the RNAs That Cause Pulmonary Fibrosis and Polycystic Kidney DiseaseUS ARMY MED RES ACQUISITION · Principal Investigator
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Apr 2022
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Mar 2025
Targeting mRNAs that Contribute to Neurological DiseaseEXPANSION THERAPEUTICS · Principal Investigator
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Apr 2022
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Mar 2025
Targeted degradation of RNAs by using small moleculesNATL INST OF HLTH NCI · Principal Investigator
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Apr 2022
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Jun 2025
Small Molecule Targeting of mRNAs Encoding Proteins relevant for Cardiovascular, Renal and Metabolic DiseasesASTRAZENECA · Principal Investigator
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Apr 2022
ACTIVE
Design of precision small molecules targeting RNA repeating transcripts to manipulate and study disease biologyNATL INST OF HLTH NINDS · Principal Investigator
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2003
Ph.D. in Biophysical ChemistryUniversity of Rochester
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1999
Master's of Science in ChemistryUniversity of Rochester
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1997
Bachelor's of Science in ChemistryUniversity of Maryland, College Park
Ezgi Hacisuleyman
- Physical Address:
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Kavli Neuroscience Postdoctoral Fellowship2021-2023 · Kavli Neural Systems Institute at The Rockefeller University
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Helen Hay Whitney Fellow2016-2019 · Helen Hay Whitney Foundation
- 2025 bioRxiv : the preprint server for biology
- 2024 Nature neuroscience
- 2021 Frontiers in integrative neuroscience
- 2021 The New England journal of medicine
- 2021 PloS one
- 2021 The New England journal of medicine
- 2016 Nature communications
- 2016 Nature communications
- 2015 Nature methods
- 2014 Nature structural & molecular biology
- 2013 Proceedings of the National Academy of Sciences of the United States of America
- 2013 eLife
- 2012 Genome biology
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Aug 2025
ACTIVE
Decoding RNA Localization and Local Translation: Key Mechanisms of Cellular Adaption and FunctionNATL INST OF HLTH NIGMS · Principal Investigator
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2010-2015
Ph.D. in Molecular Biology and BiochemistryHarvard University
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2006-2010
Bachelor of Science, Molecular Biology and GeneticsMassachusetts Institute of Technology
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2006-2010
Bachelor of Engineering, Chemical EngineeringMassachusetts Institute of Technology
Michael Harris
- Mailing Address:
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Jul 2020
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Sep 2025
Chemistry-Biology Interface Training Program at the University of FloridaNATL INST OF HLTH NIGMS · Other
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Jun 2020
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Jun 2022
NMR console upgrade for structural biology and metabolomicsNATL INST OF HLTH OD · Faculty
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Jul 2018
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Jun 2025
Specificity in Substrate Recognition and Catalysis by RNA Processing EnzymesNATL INST OF HLTH NIGMS · Principal Investigator
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Mar 2017
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Feb 2019
Mechanistic Enzymology of Phosphoryl Transfer EnzymesNATL INST OF HLTH NIGMS · Principal Investigator
Yanjun Li Ph.D.
- 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.
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UF Health Cancer Center Annual Research Showcase Poster Winner2025 · University of Florida Health Cancer Institute
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UF College of Pharmacy 38th Annual Research Showcase Poster Winner,2025 · College of Pharmacy, University of Florida
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Excellence in Instructional Redesign2025 · College of Pharmacy, University of Florida
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.
- 2025 RSC Chemical Biology
- 2024 Methods in Molecular Biology
- 2024 Journal of Controlled Release
- 2024 Scientific Reports
- 2024 Briefings in Bioinformatics
- 2024 Bioinformatics Advances
- 2023 Briefings in bioinformatics
- 2022 IEEE/ACM Transactions on Computational Biology and Bioinformatics
- 2022 Frontiers in Genetics
- 2022 PLOS Digital Health
- 2022 Journal of Chemical Information and Modeling
- 2021 Surgery
- 2020 Surgery
- 2019 2019 IEEE International Conference on Bioinformatics and Biomedicine (BIBM)
- 2018 Nature Genetics
- 2018 Nature Genetics
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Aug 2025
ACTIVE
Cell Morphology-guided, Scalable and Controllable Molecule Design via Generative AINATL INST OF HLTH NIBIB · Principal Investigator
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Aug 2024
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Aug 2025
Hijacking Post-translational Arginylation for Targeted Protein DegradationWASHINGTON UNIV SAINT LOUIS · Principal Investigator
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Jun 2024
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Dec 2025
Developing Artificial Intelligence and Machine Learning Approaches to Identify and Optimize Small Molecule Medicines Targeting Oncogenic RNAsFL DEPT OF HLTH · Co-Project Director/Principal Investigator
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Jun 2024
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Dec 2025
Enhancing Cancer Diagnosis and Treatment through Innovative Glycan- Specific Protein EngineeringFL DEPT OF HLTH · Co-Investigator
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Dec 2023
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Dec 2024
ExoTarget Platform as a programmable delivery systemNATL INST OF HLTH · Co-Investigator
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Jun 2023
ACTIVE
UF Health Cancer Center Support GrantNATL INST OF HLTH NCI · Project Manager
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2021
Ph.D. (Computer Science)University of Florida
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2015
M.S. (Information, Production and Systems Engineering)Waseda University
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2015
M.S. (Communication and Information System)University of Electronic Science and Technology of China
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2012
B.S. (Information Engineering)University of Electronic Science and Technology of China
Chenglong Li Ph.D.
- 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.
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
- 2025 EXCLI Journal
- 2025 ACS Omega
- 2025 Journal of the American Chemical Society
- 2025 ACS Medicinal Chemistry Letters
- 2025 ACS Chemical Biology
- 2025 Processes
- 2024 Organic & Biomolecular Chemistry
- 2024 Briefings in Bioinformatics
- 2024 Clinical Pharmacology & Therapeutics
- 2024 ACS Medicinal Chemistry Letters
- 2024 Journal of Medicinal Chemistry
- 2024 Journal of Medicinal Chemistry
- 2024 Journal of Medicinal Chemistry
- 2024 Journal of Medicinal Chemistry
- 2023 Molecules
- 2023 Clinical Cancer Research
- 2023 Bioorganic & Medicinal Chemistry Letters
- 2022 Molecular cancer therapeutics
- 2022 FASEB journal : official publication of the Federation of American Societies for Experimental Biology
- 2022 Journal of chemical information and modeling
- 2022 Cell death discovery
- 2022 Communications Biology
- 2022 Bioorganic & medicinal chemistry
- 2021 European journal of pharmacology
- 2021 JCI insight
- 2021 Scientific reports
- 2021 Journal of natural products
- 2021 Oxidative medicine and cellular longevity
- 2021 PLOS One
- 2021 ACS chemical neuroscience
- 2020 iScience
- 2020 IEEE/ACM transactions on computational biology and bioinformatics
- 2020 Journal of cellular and molecular medicine
- 2020 Frontiers in pharmacology
- 2020 Journal of medicinal chemistry
- 2020 Breast cancer research and treatment
- 2020 Oncogene
- 2020 Chemical science
- 2020 Journal of virology
- 2020 Life sciences
- 2020 Cancer research
- 2020 Frontiers in chemistry
- 2019 Journal of experimental & clinical cancer research : CR
- 2019 Cancer science
- 2019 Clinical and experimental immunology
- 2019 Current cancer drug targets
- 2019 Analytical biochemistry
- 2018 International journal of oncology
- 2018 Atherosclerosis
- 2018 Neuro-oncology
- 2018 Journal of medicinal chemistry
- 2017 The Journal of Immunology
- 2017 Scientific reports
- 2017 Nature biomedical engineering
- 2017 Bioorganic & medicinal chemistry
- 2017 Journal of extracellular vesicles
- 2017 PloS one
- 2017 International Journal of Oncology
- 2017 Biochemical and Biophysical Research Communications
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Sep 2024
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Aug 2025
AI-directed discovery of heparanase inhibitorsNATL INST OF HLTH NIDDK · Principal Investigator
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Jul 2024
ACTIVE
Dual Inhibition of PARP and IL-6 as a Novel Therapeutic Approach for BRCA-Mutated Triple-Negative Breast CancerUS ARMY MED RES ACQUISITION · Principal Investigator
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Jun 2024
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Dec 2025
Developing Artificial Intelligence and Machine Learning Approaches to Identify and Optimize Small Molecule Medicines Targeting Oncogenic RNAsFL DEPT OF HLTH · Co-Project Director/Principal Investigator
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Feb 2024
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Jan 2026
Dual inhibition of PARP and STAT3 as a novel therapeutic approach for triple-negative breast cancerUNIV OF MARYLAND BALTIMORE · Principal Investigator
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Sep 2023
ACTIVE
Mechanisms of Metal Ion Homeostasis of Oral StreptococciNATL INST OF HLTH NIDCR · Co-Investigator
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Jul 2023
ACTIVE
Developing YAP/TEAD Inhibitors for Novel Nash-Associated Hepatocellular Carcinoma TherapeuticsUS ARMY MED RES ACQUISITION · Principal Investigator
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May 2023
ACTIVE
Development of high-affinity and selective ligands as a pharmacological tool for the Dopamine D4 receptor (D4R) subtype variantsHIGH POINT UNIVERSITY · Principal Investigator
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Aug 2022
ACTIVE
Integrative Multidisciplinary Discovery Platform to Unlock Marine Natural Products Therapeutic OpportunitiesNATL INST OF HLTH NIGMS · Co-Investigator
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Feb 2022
–
Jan 2025
Computationally designed IL-10 mutants for the treatment of inflammatory bowel diseasesUS ARMY MED RES ACQUISITION · Co-Investigator
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Jan 2022
–
Jan 2023
HEC Bench Fee – Tahira NoorHIGHER EDUCATION COMMISSION PAKISTAN · Principal Investigator
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Jan 2021
–
Dec 2022
Artificial Intelligence Research Catalyst FundUF RESEARCH · Project Manager
-
Jul 2020
–
Sep 2025
Chemistry-Biology Interface Training Program at the University of FloridaNATL INST OF HLTH NIGMS · Principal Investigator
-
Sep 2019
–
Aug 2023
QLCI-CG: Conceptualization of the Institute for Quantum Biology on Quantum ComputersNATL SCIENCE FOU · Co-Project Director/Principal Investigator
-
Sep 2019
–
Jan 2025
Novel Targeted Anticancer Agents from Marine CyanobacteriaNATL INST OF HLTH NCI · Other
-
Sep 2018
–
Aug 2022
Mechanistic studies of gamma-glutamyl transpeptidase inhibition: A novel approach to modulating serum levels of cysteineUNIV OF OKLAHOMA NORMAN · Principal Investigator
-
Jun 2017
–
May 2023
Role and targeting of PRMT5 in prostate cancerPURDUE UNIV · Principal Investigator
-
Sep 2016
–
Jan 2018
Repositioning Bazedoxifene as a novel IL-6/GP130 inhibitor for sarcoma therapyUNIV 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 therapyNATL INST OF HLTH NINDS · Principal Investigator
-
Aug 2016
–
May 2021
A novel STAT3-selective inhibitor for medulloblastoma therapyUNIV OF MARYLAND COLLEGE PARK · Principal Investigator
-
Published
February 2019
IL-6 Inhibitors and Methods of Treatment#US-2021-0206718-A1
-
2000
Ph.D.Cornell University
-
1988
M.Sc.Beijing University
-
1985
B.Sc.Beijing University
Zhonglin Liu
- Physical Address:
Liu lab focuses on developing new chemical modalities, chemical probes, and ABPP platforms to discover novel therapeutic approaches for human diseases.
- Chemical biology
- Genetics and Genomics
- Medicinal chemistry
- Molecular Biology
- proteomics
- 2025 Nature Chemical Biology
-
2024
Proteomic Ligandability Maps of Spirocycle Acrylamide Stereoprobes Identify Covalent ERCC3 DegradersJournal of the American Chemical Society
- 2024 Nature
- 2023 Angewandte Chemie
- 2021 Journal of the American Chemical Society
- 2020 Chemical Science
- 2019 Angewandte Chemie International Edition
- 2019 Chem
- 2019 Organic Letters
- 2018 Journal of the American Chemical Society
- 2017 Organic Letters
- 2017 Journal of the American Chemical Society
- 2017 Journal of the American Chemical Society
- 2017 Angewandte Chemie International Edition
-
Jun 2023
ACTIVE
UF Health Cancer Center Support GrantNATL INST OF HLTH NCI · Project Manager
-
2012-2017
Ph.D.Shanghai Institute of Organic Chemistry, CAS
-
2008-2012
B.S.Nanchang University
Luiz Passalacqua
- Mailing Address:
- Physical Address:
- 2025 eLife
- 2024 eLife
- 2024 RNA (New York, N.Y.)
- 2024 Nucleic acids research
- 2024 The Journal of biological chemistry
- 2023 Nature communications
- 2023 Nature
- 2022 The Journal of biological chemistry
- 2020 eLife
Sathyanarayanan V Puthanveettil Ph.D.
- 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.
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CAREER Award2015 · National Science Foundation
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Whitehall Foundation Award2012 · Whitehall Foundation
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Young Investigator Award2011 · Margaret Q. Landenberger Research Foundation
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TSRI Nomineee for Alfred Sloan Award, Searle Scholar Award2011 · —
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Columbia University Nominee for Blavatnik Award2010 · New York Academy of Sciences
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Recognition for work on Kinesin transported RNAs as “HOT TOPIC” for Neuroscience 20092009 · Society For Neuroscience (SFN)
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Cold Spring Harbor Laboratory Registration Waiver2009 · —
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Prize for Innovative Research Ideas2003 · The National Neurofibromatosis Foundation and International Neurofibromatosis Research Foundation
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Travel Grant2000 · American Society for Gravitational and Space Biology (ASGSB)
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Travel Award2000 · Washington State University (WSU) Graduate School
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Gordon Research Conference Chair’s Funds for Registration Fees2000 · —
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Cold Spring Harbor Laboratory Registration waiver1997 · —
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National Merit Scholarship1984-1991 · Department of Education, India
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.
- 2024 Nature communications
- 2024 Scientific reports
- 2024 Aging cell
- 2023 eNeuro
- 2022 Methods in molecular biology (Clifton, N.J.)
- 2022 Molecular and cellular neurosciences
- 2021 Cells
- 2021 Science advances
- 2021 RNA biology
- 2021 Cell reports
- 2021 Molecular brain
- 2020 Science advances
- 2020 Frontiers in cellular neuroscience
- 2019 Cell reports
- 2019 Neurobiology of learning and memory
- 2018 Molecular neuropsychiatry
- 2018 Proceedings of the National Academy of Sciences of the United States of America
- 2018 Scientific reports
- 2017 Biological psychiatry. Cognitive neuroscience and neuroimaging
- 2015 Frontiers in cellular neuroscience
- 2015 Neuropsychopharmacology : official publication of the American College of Neuropsychopharmacology
- 2015 Frontiers in cellular neuroscience
- 2014 Frontiers in aging neuroscience
- 2014 Journal of visualized experiments : JoVE
- 2014 Frontiers in genetics
- 2014 PloS one
- 2014 Assay and drug development technologies
- 2014 Proceedings of the National Academy of Sciences of the United States of America
- 2013 Nature structural & molecular biology
- 2013 Proceedings of the National Academy of Sciences of the United States of America
- 2013 Molecular bioSystems
- 2013 RNA biology
- 2013 PloS one
- 2013 BMC genomics
- 2012 Proceedings of the National Academy of Sciences of the United States of America
- 2012 Proceedings of the National Academy of Sciences of the United States of America
- 2012 Translational neuroscience
- 2011 Neuron
- 2009 Neuron
- 2008 Neuron
- 2008 Cell
- 2007 Proceedings of the National Academy of Sciences of the United States of America
- 2006 Nucleic acids research
- 2006 Cell
-
May 2025
ACTIVE
Determining the Molecular Arrangement of GABABR-GIRK Signaling CascadesNATL INST OF HLTH NIDA · Other
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Mar 2025
–
Oct 2025
Defining the mechanism of PTCHD1 and membrane cholesterol in the regulation of the mu-opioid receptorNATL INST OF HLTH NIGMS · Other
-
Jan 2025
ACTIVE
Cell specific & compartment specific changes in translation during long term memory storageNATL INST OF HLTH NIMH · Other
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Jan 2024
–
Jan 2025
Molecular basis for cholesterol regulation of Kir2.1 in cardiac arrhythmiaAMER HEART ASSOCIATION · Other
-
Dec 2023
–
Nov 2025
Analysis of RNAs targeted to the long-distance projections of ventral hippocampusNATL INST OF HLTH NIMH · Principal Investigator
-
Sep 2022
–
Aug 2024
Novel role of mitotic kinesin KIF11 in structural plasticity and memoryNATL INST OF HLTH NIMH · Other
-
Jun 2022
–
May 2025
Assessing effects of manipulation of expression of kinesins in a mouse model of Alzheimer's diseaseNATL INST OF HLTH NIA · Principal Investigator
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Apr 2022
–
Jun 2023
Assessing the role of circRNAs in memory consolidationNATL INST OF HLTH NIMH · Principal Investigator
-
Apr 2022
–
Aug 2024
The role of bidirectional transport of lysosome-related organelles in learning and memory storageNATL INST OF HLTH NIMH · Principal Investigator
-
Apr 2022
–
Oct 2024
Dynein-Dynactin Complex Mediated Retrograde Transport during Long-term Memory StorageNATL INST OF HLTH NIMH · Principal Investigator
-
Apr 2022
–
Jul 2025
Transcriptomic Mechanisms of Formation and Persistence of Synapse Specific Long-Term MemoryNATL INST OF HLTH NIMH · Principal Investigator
-
Apr 2022
–
Feb 2025
Bidirectional Transport of Lysosome Related Organelles during Synapse Maintenance and PlasticityNATL SCIENCE FOU · Principal Investigator
-
2001
Ph.D. in Biochemistry/BiophysicsWashington State University
-
1994
Master's of Science in BiotechnologyTamil Nadu Agricultural University
-
1991
Bachelor's of Science in AgricultureKerala Agricultural University
Laura P W Ranum
- 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.
- 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
- 2026 Cell Reports
- 2025 Proceedings of the National Academy of Sciences
-
2023
Choroid plexus mis-splicing and altered cerebrospinal fluid composition in myotonic dystrophy type 1Brain
- 2023 Human Genetics
- 2022 Current Opinion in Neurobiology
- 2022 Neuron
-
2021
CCG•CGG interruptions in high penetrance SCA8 families increase RAN translation and protein toxicityBioRx
- 2021 Human Molecular Genetics
- 2021 Current Opinion in Neurology
-
2021
CCG•CGG interruptions in high‐penetrance SCA8 families increase RAN translation and protein toxicityEMBO Molecular Medicine
- 2020 Neuron
- 2020 Nature communications
-
2020
Metformin inhibits RAN translation through PKR pathway and mitigates disease in C9orf72 ALS/FTD miceProceedings of the National Academy of Sciences
-
2020
Survival and Motor Phenotypes in FVB C9-500 ALS/FTD BAC Transgenic Mice Reproduced by Multiple Labs.Neuron
- 2020 Human Molecular Genetics
- 2019 Laboratory investigation; a journal of technical methods and pathology
- 2019 Annual review of neuroscience
- 2018 Proceedings of the National Academy of Sciences of the United States of America
- 2018 Neurobiology of disease
- 2018 Trends in neurosciences
- 2018 The EMBO Journal
- 2018 The Journal of biological chemistry
- 2018 Cold Spring Harbor perspectives in biology
- 2017 Current opinion in genetics & development
- 2017 Neuron
- 2017 PloS one
- 2017 Neuron
- 2016 Neuron
- 2015 Stem cells (Dayton, Ohio)
- 2015 Cell reports
- 2015 Neuron
- 2014 Current opinion in genetics & development
- 2014 Neurobiology of aging
- 2013 Embo Molecular Medicine
- 2013 Acta neuropathologica
- 2013 Human molecular genetics
- 2013 Proceedings of the National Academy of Sciences of the United States of America
- 2012 Handbook of clinical neurology
- 2012 Archives of neurology
- 2012 Neurology
- 2012 Archives of neurology
- 2012 Human molecular genetics
- 2012 Handbook of clinical neurology
- 2011 Proceedings of the National Academy of Sciences of the United States of America
- 2011 PloS one
- 2011 European journal of human genetics : EJHG
- 2010 The Journal of cell biology
- 2010 Human molecular genetics
- 2010 Rinsho shinkeigaku = Clinical neurology
- 2009 PloS one
- 2009 PLoS genetics
- 2008 DNA repair
- 2007 Archives of neurology
- 2006 Annual review of neuroscience
- 2006 Nature genetics
- 2006 Journal of medical genetics
- 2006 Human molecular genetics
- 2006 Nature genetics
- 2005 Neuromuscular disorders : NMD
- 2005 Current neurology and neuroscience reports
- 2004 American journal of human genetics
- 2004 American journal of human genetics
- 2004 Trends in genetics : TIG
- 2004 The Journal of biological chemistry
- 2003 Methods in molecular biology (Clifton, N.J.)
- 2003 American journal of human genetics
- 2002 Current opinion in genetics & development
- 2002 Current neurology and neuroscience reports
- 2001 Science (New York, N.Y.)
- 2000 Nature genetics
- 2000 Human molecular genetics
- 1999 Neuromuscular disorders : NMD
- 1999 Nature genetics
- 1999 Human molecular genetics
- 1999 Neurology
-
Sep 2025
ACTIVE
Molecular effects of metformin, PKR and TBI on C9orf72 ALS/FTDNATL 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 opportunitiesNATL INST OF HLTH NINDS · Principal Investigator
-
May 2025
ACTIVE
Proof of concept testing of an anti-GA immunotherapy for C9orf72 ALS/FTDLATUS BIO · Principal Investigator
-
Dec 2024
–
Nov 2025
10th International Unstable Microsatellites and Human Disease MeetingNATL INST OF HLTH NINDS · Principal Investigator
-
Aug 2024
ACTIVE
Senator Paul D. Wellstone Muscular Dystrophy Specialized Research CenterNATL INST OF HLTH NINDS · Faculty
-
Apr 2024
ACTIVE
Contribution of Repeat Associated Non-AUG (RAN) Proteins in Friedreich's and Other Hereditary AtaxiasUS ARMY MED RES ACQUISITION · Principal Investigator
-
Sep 2023
ACTIVE
Safety of Metformin in C9orf72 ALS: Effects on RAN Proteins, Breathing, Imaging, and Metabolomic Outcome MeasuresUS 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 modelsUS ARMY MED RES ACQUISITION · Principal Investigator
-
May 2023
ACTIVE
Anti-RAN targeting immunotherapy for C9orf72 ALS and genetically unknown RAN-positive sporadic ALSUS ARMY MED RES ACQUISITION · Principal Investigator
-
Mar 2023
–
Feb 2025
Repeat associated non-AUG (RAN) proteins in FA: contribution to disease and therapeutic opportunitiesFRIEDREICHS ATAXIA RESEARCH ALLIANCE · Other
-
Feb 2023
ACTIVE
An RNA-based nanoparticle vaccine approach for the prevention and treatment of C9orf72 ALS/FTDUS ARMY MED RES ACQUISITION · Principal Investigator
-
Dec 2022
–
Nov 2024
Safety & therapeutic potential of metformin for C9orf72 ALSAMYOTROPHIC LATERAL SCLEROSIS ASSOC · Principal Investigator
-
Sep 2022
–
Aug 2025
Molecular effects of metformin, PKR and TBI on C9orf72 ALS/FTDNATL INST OF HLTH NINDS · Principal Investigator
-
Jul 2022
ACTIVE
Identifying and targeting novel repeat associated non-AUG (RAN) proteins in sporadic ALSUS 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 opportunitiesNATL INST OF HLTH NINDS · Principal Investigator
-
Jan 2022
–
Dec 2023
Characterization of a Novel BAC Transgenic Mouse Model for DM2MYOTONIC DYSTROPHY FOU · Other
-
Aug 2021
–
Jul 2023
Using mitochondrial Ca2+ uptake as a therapeutic target for ALSNATL INST OF HLTH NINDS · Co-Investigator
-
Jul 2021
ACTIVE
Contribution of RAN proteins to HD, SCA3 other CAG.CTG expansion diseasesNATL INST OF HLTH NINDS · Principal Investigator
-
Mar 2021
–
Feb 2022
RAN proteins in sporadic ataxias and SCA8: diagnostic tools and therapeutic potential of metforminNATL ATAXIA FOUNDATION · Other
-
Sep 2020
–
Jun 2023
Identifying and understanding the role of repeat RNAs and RAN proteins in Alzheimer's diseaseNATL 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 modelJOHNS HOPKINS UNIVERSITY · Principal Investigator
-
Jan 2020
–
Jan 2023
Provenance Initiatives Fund at the Center for NeuroGeneticsUF FOUNDATION · Principal Investigator
-
Nov 2019
–
Oct 2021
C9ORF72 ALS: Endobody vaccine for the treatment of C9 ALS/FTDAMYOTROPHIC LATERAL SCLEROSIS ASSOC · Other
-
Sep 2019
–
Sep 2022
Therapeutic Potential of Metformin, an FDA-approved drug, and PKR Targeting for C9orf72 ALSUS ARMY MED RES AND DEVELOPMENT COMMAND · Principal Investigator
-
Dec 2018
–
Jan 2021
Presymptomatic biomarkers of C9-ALS/FTDAMYOTROPHIC LATERAL SCLEROSIS ASSOC · Principal Investigator
-
Sep 2018
–
Apr 2020
Assessment of endobody vaccine for effectiveness and ability to generate C9 RAN antibodiesUNITED NEUROSCIENCE LIMITED · Principal Investigator
-
Aug 2018
–
Feb 2021
C9ORF72 ALS: Proof of biology for antibody treatment in C9-ALS mouse modelBIOGEN MA INC · Principal Investigator
-
Aug 2018
–
Jul 2021
The contribution of RAN proteins to C9ORF72 ALS/FTDMUSCULAR DYSTROPHY ASSO · Principal Investigator
-
Feb 2018
–
Feb 2020
Therapeutic potential of CRISPR-Cas9 in C9ORF72-repeat deletion strategy in C9ORF72 BAC transgenic miceCRISPR THERAPEUTICS AG · Principal Investigator
-
Jan 2018
–
Dec 2019
Molecular characterization of RNA and RAN protein effects in DM2WYCK FOUNDATION · Other
-
Jul 2017
–
Jun 2022
Neurodegenerative disease researchUF FOUNDATION · Principal Investigator
-
Jul 2017
–
Dec 2024
University of Florida/National Ataxia Foundation Brain Donation ProgramNATL 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 miceTARGET ALS FOUNDATION · Principal Investigator
-
Jan 2017
–
Jun 2020
Nuclear Export Inhibitor KPT-350 for C9orf72 and Sporadic ALSTARGET ALS FOUNDATION · Principal Investigator
-
Jan 2017
–
Jun 2020
Developing human-derived antibodies to target dipeptide-repeat protein toxicity in C9orf72 diseaseTARGET 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 ALSAMYOTROPHIC LATERAL SCLEROSIS ASSOC · Other
-
Aug 2016
–
Jul 2018
Molecular characterization & antibody therapy in a novel C9orf72 BAC mouse modelMUSCULAR 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 ModelsNEURIMMUNE AG · Principal Investigator
-
Mar 2016
–
Apr 2021
neurodegenerative disease researchUF FOUNDATION · Principal Investigator
-
Jan 2016
–
Jul 2022
Molecular Genetics of the G4C2 expansion mutation in ALS/FTDUF FOUNDATION · Principal Investigator
-
Jan 2016
–
Jun 2023
Molecular genetic characterization of SCA8UF FOUNDATION · Principal Investigator
-
Jan 2016
–
Dec 2016
ASO targeting of bidirectional transcripts and RAN translation in SCA8NATL ATAXIA FOUNDATION · Principal Investigator
-
Dec 2015
–
Dec 2020
Brainstorming microsatellite expansion diseases symposiumUF FOUNDATION · Principal Investigator
-
Aug 2015
–
Jul 2023
Molecular Genetic Characterization of SCA8NATL INST OF HLTH NINDS · Principal Investigator
-
Jan 2015
–
Jan 2018
MBI Research Program MGOLD1UF FOUNDATION · Principal Investigator
-
Sep 2013
–
Jun 2019
Myotonic Dystrophy: Molecular Pathophysiology and CNS EffectsNATL 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 NeuroGeneticsUF FOUNDATION · Principal Investigator
-
May 2013
–
Apr 2016
Repeat associated non-ATG (RAN) Translation in C9ORF72 ALS/FTDCOLUMBIA UNIVERSITY · Principal Investigator
-
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
- 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.
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
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2026
Cross‐Species Insights Into Gamma Herpesvirus Transcriptomes: Long‐Read and Multi‐Omics PerspectivesJournal of Medical Virology
- 2025 Exploration of Targeted Anti-tumor Therapy
- 2024 Health expectations : an international journal of public participation in health care and health policy
- 2024 Journal of the American Heart Association
- 2024 Scientific Reports
- 2024 Nucleic Acids Research
- 2023 bioRxiv : the preprint server for biology
- 2023 Journal of Virology
- 2023 mBio
- 2023 mSphere
- 2023 Frontiers in oncology
- 2023 Molecular biology and evolution
- 2023 Bioinformatics
- 2022 Scientific reports
- 2022 Viruses
- 2021 Cancers
- 2021 Journal of virology
- 2021 PLOS Pathogens
- 2021 RNA (New York, N.Y.)
- 2021 Current protocols
- 2021 PLOS Genetics
- 2020 Cell host & microbe
- 2020 Drug metabolism and disposition: the biological fate of chemicals
- 2019 Journal of virology
- 2019 mBio
- 2019 PLOS Pathogens
- 2019 PLoS pathogens
- 2019 Gene therapy
- 2018 Journal of virology
- 2018 Proceedings of the National Academy of Sciences of the United States of America
- 2018 PLoS pathogens
- 2018 Nucleic acids research
- 2018 Non-coding RNA
- 2018 Trends in cancer
- 2017 PLOS Pathogens
- 2016 mBio
- 2016 Viruses
- 2016 Oncotarget
- 2016 PLoS pathogens
- 2016 mSphere
- 2016 Oncotarget
- 2015 Journal of virology
- 2015 Journal of virology
- 2015 Journal of virology
- 2015 PLoS pathogens
- 2015 PLoS pathogens
- 2014 Computational and structural biotechnology journal
- 2014 Critical reviews in eukaryotic gene expression
- 2014 mBio
- 2014 PLoS pathogens
- 2014 Nucleic acids research
- 2014 Science (New York, N.Y.)
- 2014 BMC genomics
- 2014 Viruses
- 2014 Oncotarget
- 2013 Nucleic acids research
- 2013 Current opinion in virology
- 2013 Journal of medical virology
- 2013 Journal of virology
- 2012 Journal of virology
- 2012 PLoS pathogens
- 2011 Methods in molecular biology (Clifton, N.J.)
- 2011 Biochimica et biophysica acta
- 2011 Journal of virology
- 2010 PLoS pathogens
- 2010 Genes & development
- 2010 Journal of virology
- 2010 Current opinion in microbiology
- 2010 The Journal of general virology
- 2009 Glia
- 2009 Journal of virology
- 2009 Trends in microbiology
-
2008
KSHV LANA inhibits TGF-beta signaling through epigenetic silencing of the TGF-beta type II receptor.Blood
- 2007 The Journal of infectious diseases
- 2007 PLoS pathogens
- 2007 Journal of virology
- 2007 Journal of virology
- 2006 Journal of virology
- 2006 Inhalation toxicology
- 2005 The Journal of biological chemistry
-
2005
Characterization of the minimal replicator of Kaposi's sarcoma-associated herpesvirus latent origin.Journal of virology
- 2005 Journal of virology
- 2004 The Journal of biological chemistry
- 2002 The Journal of biological chemistry
- 2002 Journal of virology
-
Jun 2025
ACTIVE
The Florida Summer Training in Research and Oncology for the Next Generation of Researchers (STRONGER) ProgramNATL INST OF HLTH NCI · Other
-
Jun 2023
ACTIVE
UF Health Cancer Center Support GrantNATL 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 outcomesUS ARMY MED RES ACQUISITION · Other
-
Jun 2022
ACTIVE
Noncoding RNAs in gamma-Herpesvirus Biology and AIDS MalignanciesNATL INST OF HLTH NCI · Principal Investigator
-
Jan 2022
ACTIVE
MicroRNA turnover induced by target RNAs in Colorectal CancerAMERICAN CANCER SOCIETY · Co-Investigator
-
Jan 2019
–
Oct 2020
UFF F006200 Innes ProfessorshipUF FOUNDATION · Principal Investigator
-
Sep 2018
–
Jun 2024
The Role of H3.3 histone variant in the pathogenesis of oral Kaposi's SarcomaNATL INST OF HLTH NIDCR · Principal Investigator
-
Feb 2017
–
Jan 2022
Noncoding RNAs in gamma-Herpesvirus Biology and AIDS MalignanciesNATL INST OF HLTH NCI · Principal Investigator
-
Jul 2015
–
Jun 2024
Florida Academic Cancer Center Alliance Seed GrantsUF 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 ProgramUF HEALTH SHANDS HOSPITAL · Project Manager
-
Jan 2015
–
Dec 2020
Role of MHV68 miRNAs in latency and pathogenesisNATL 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 ProgramUF HEALTH SHANDS HOSPITAL · Project Manager
-
Jul 2014
–
Jun 2017
Histone variant H3.3 and KSHV LANA in the pathogenesis of oral Kaposis sarcomaNATL INST OF HLTH NIDCR · Principal Investigator
-
Aug 2013
–
Aug 2016
Identification of KSHV miRNA targets in endothelial cells and characterization miRNA knockout virusesNATL INST OF HLTH · Principal Investigator
-
Dec 2012
–
Jan 2019
Studying the role of KSHV-encoded miRNAsNATL INST OF HLTH NCI · Principal Investigator
-
Mar 2011
–
Feb 2017
LANA and Cellular Gene ExpressionNATL INST OF HLTH NCI · Principal Investigator
-
Mar 2006
–
May 2023
Davis Cancer Researach EquipmentUF FOUNDATION · Project Manager
-
1989-1993
PhD, Retrovirology/Foamy VirusesAlbert-Ludwigs University
-
1984-1989
MS, BiologyAlbert-Ludwigs University
Elias Sayour MD, PhD
- 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.
-
Hematology-OncologyAmerican Board of Pediatrics
-
PediatricsAmerican Board of Pediatrics
- Neurosurgery
- Pediatrics
- Pediatric Hematology-Oncology
- Brain tumor – children
- Immunotherapeutics for CNS malignancies
- Pediatric brain tumors
- 2026 Nature Cancer
- 2025 Advanced science (Weinheim, Baden-Wurttemberg, Germany)
- 2025 Molecular oncology
- 2025 Molecular Therapy
- 2025 Journal of Clinical Investigation
- 2025 Nature Biomedical Engineering
- 2025 Human Vaccines & Immunotherapeutics
- 2025 Nature
- 2025 Nature
- 2025 Journal for ImmunoTherapy of Cancer
- 2024 Journal of hematology & oncology
- 2024 Molecular therapy. Methods & clinical development
- 2024 Cell
- 2024 Nature Reviews Clinical Oncology
- 2024 Neurosurgical focus
- 2023 bioRxiv : the preprint server for biology
- 2023 bioRxiv : the preprint server for biology
- 2023 medRxiv : the preprint server for health sciences
- 2023 Biointerphases
- 2023 Neuro-oncology
- 2023 Cancers
- 2023 Frontiers in oncology
- 2023 Neoplasia (New York, N.Y.)
- 2023 Journal of applied statistics
- 2023 Acta Biomaterialia
- 2022 Nature cancer
- 2022 Cells
-
2022
Fusobacterium is enriched in oral cancer and promotes induction of programmed death-ligand 1 (PD-L1)Neoplasia
- 2022 Neurologia medico-chirurgica
- 2021 Translational Oncology
- 2021 Veterinary immunology and immunopathology
- 2021 NeuroMolecular Medicine
- 2021 Frontiers in immunology
- 2020 American journal of cancer research
- 2020 Cancer research
- 2020 Journal of Neuro-Oncology
- 2019 Molecular therapy : the journal of the American Society of Gene Therapy
- 2019 Neuro-oncology
- 2019 Journal of hematology & oncology
- 2019 Nature communications
- 2019 Veterinary immunology and immunopathology
- 2019 Journal for immunotherapy of cancer
- 2019 ACS nano
- 2018 Neuro-oncology
- 2018 Frontiers in molecular neuroscience
- 2018 Nano Letters
- 2018 International journal of molecular sciences
- 2018 The EMBO journal
- 2017 Journal of Immunology Research
- 2017 Oncoimmunology
- 2017 Journal of neurosurgery
- 2017 Brain sciences
- 2017 Oncoimmunology
- 2017 Frontiers in immunology
- 2016 Cancer immunology, immunotherapy : CII
- 2016 Clinical cancer research : an official journal of the American Association for Cancer Research
- 2016 Histopathology
- 2016 Pediatric blood & cancer
- 2015 Oncoimmunology
- 2015 Cancer immunology research
- 2015 Cancer immunology, immunotherapy : CII
- 2015 Journal for immunotherapy of cancer
- 2015 Pediatric blood & cancer
- 2014 PloS one
- 2013 Cancer immunology, immunotherapy : CII
-
Dec 2025
ACTIVE
Unlocking CAR T cells Activity Against Metastatic Osteosarcoma Using Systemic RNA VaccinationHYUNDAI HOPE ON WHEELS · Other
-
Nov 2025
ACTIVE
Enhancing Immunotherapy Efficacy in NSCLC through Tumor Antigen-Targeted RNA NanoparticlesFL 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 TissueNATL PEDIATRIC CANCER FOU · Principal Investigator
-
Sep 2025
ACTIVE
Overcoming metastatic spread of osteosarcoma with RNA loaded nanoparticlesNATL INST OF HLTH NCI · Principal Investigator
-
Jun 2025
ACTIVE
The Florida Summer Training in Research and Oncology for the Next Generation of Researchers (STRONGER) ProgramNATL 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 DMGDIPG DMG RESEARCH FUNDING ALLIANCE · Principal Investigator
-
May 2025
ACTIVE
ReSubmission R01 Role of bacterial-derived small molecules in immunotherapyNATL INST OF HLTH NCI · Co-Investigator
-
Feb 2025
–
Jan 2026
Multilamellar RNA aggregates targeting diffuse midline glioma, including diffuse intrinsic pontine gliomaCHILDRENS NATIONAL RESEARCH INSTITUTE · Principal Investigator
-
Oct 2024
ACTIVE
Nivolumab and DAY101 for the treatment of newly diagnosed or recurrent craniopharyngioma in children and young adultsUNIV OF CALIFORNIA SAN FRANCISCO · Principal Investigator
-
Sep 2024
ACTIVE
Targeted RNA-Lipid Particle Aggregates in Pediatric Oncology: A Novel Therapeutic Strategy for Fibrolamellar Hepatocellular CarcinomaHYUNDAI HOPE ON WHEELS · Other
-
Sep 2024
–
Sep 2024
Task Order Number: 15349sc PNOC COMPUNIV OF CALIFORNIA SAN FRANCISCO · Principal Investigator
-
Sep 2024
ACTIVE
Task Order Number: 15349sc PNOC COMPUNIV OF CALIFORNIA SAN FRANCISCO · Principal Investigator
-
Jul 2024
ACTIVE
Phase 2 Trial of G207 + 5 Gy Radiation for Children with High-Grade GliomasST 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 MBsJOHNS HOPKINS ALL CHILDRENS HOSPITAL · Principal Investigator
-
Jul 2024
ACTIVE
Combining Epigenetic Therapy and Immunotherapy to Target Group 4 MedulloblastomaCHILDRENS RESEARCH INSTITUTE · Co-Investigator
-
May 2024
–
Jul 2024
Pediatric Oncology Student Training (POST) ProgramALEXS LEMONADE STAND FOUNDATION · Principal Investigator
-
May 2024
ACTIVE
Combining Epigenetic Therapy and Immunotherapy to Target Group 4 MedulloblastomaCHILDRENS RESEARCH INSTITUTE · Project Manager
-
May 2024
ACTIVE
B7-H3-CAR iNKT Therapy for High Risk MedulloblastomaNATL PEDIATRIC CANCER FOU · Principal Investigator
-
Apr 2024
ACTIVE
Targeting Glycogen Metabolism in Ewing's Sarcoma: Diagnostic, Prognostic, and Therapeutic ApplicationsNATL INST OF HLTH NCI · Co-Investigator
-
Feb 2024
–
Jan 2026
RNA-nanoparticle (RNA-NP) vaccine for ARMSKATE AMATO FOUNDATION · Other
-
Jul 2023
–
Jun 2024
Studying the impact of RNA-nanoparticle vaccine on immune response to DIPG/DMGDIPG DMG RESEARCH FUNDING ALLIANCE · Other
-
Jul 2023
–
Feb 2025
Nanoparticle vaccines targeting metastatic osteosarcomaOSTEOSARCOMA 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 TumorsH LEE MOFFITT CANCER CTR & RES INST · Principal Investigator
-
Jun 2023
ACTIVE
UF Health Cancer Center Support GrantNATL INST OF HLTH NCI · Other
-
Feb 2023
ACTIVE
An RNA-based nanoparticle vaccine approach for the prevention and treatment of C9orf72 ALS/FTDUS 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 gliomaALEXS 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 OsteosarcomaPEDIATRIC CANCER RESEARCH FOU · Other
-
Dec 2022
–
Jun 2025
Establishment of 3D metastatic osteosarcoma organoid model to accelerate combination immunotherapyHYUNDAI 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 osteosarcomaNATL PEDIATRIC CANCER FOU · Other
-
Sep 2022
–
Aug 2025
Overcoming metastatic spread of osteosarcoma with RNA loaded nanoparticlesNATL INST OF HLTH NCI · Principal Investigator
-
Aug 2022
ACTIVE
A phase I study of RNA-lipid particle vaccines for newly-diagnosed glioblastoma, IND19304 05/21/2020US FOOD AND DRUG ADMN · Principal Investigator
-
Jul 2022
ACTIVE
Leveraging RNA-lipid nanoparticle vaccines to induce immune response in metastatic pulmonary osteosarcomaTHE V FOU FOR CANCER RESEARCH · Co-Investigator
-
May 2022
–
May 2025
Patterns of resistance to initial therapeutic treatment in Ewings SarcomaHematology/Oncology Pharmacy Asso Fou · Co-Investigator
-
Apr 2022
–
Mar 2025
Unlocking CAR T cell efficacy against osteosarcoma using adjuvant RNA vaccineFL 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 therapyFL 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 GliomasUNIV 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 GliomasUNIV OF CALIFORNIA SAN FRANCISCO · Principal Investigator
-
Jan 2022
–
Jun 2023
Development of RNA Loaded Nanoparticles for Children with Refractory Solid TumorsH LEE MOFFITT CANCER CTR & RES INST · Principal Investigator
-
Jan 2022
–
Dec 2024
Development of RNA Loaded Nanoparticles for Children with Refractory Solid TumorsNATL PEDIATRIC CANCER FOU · Principal Investigator
-
Oct 2021
–
Apr 2023
Pan-Neoantigen Targeted RNA-nanoparticles for Recurrent Pulmonary OsteosarcomaCHANCE FOR LIFE FOUNDATION · Principal Investigator
-
Sep 2021
–
Aug 2024
Immune correlate studies in pediatric high-grade glioma patients receiving RNA nanoparticle vaccinesTEAM JACK FOUNDATION · Principal Investigator
-
Jul 2021
–
Aug 2022
Immunotherapeutic targeting of osteosarcoma with nanoparticle vaccinesRally Foundation · Principal Investigator
-
Apr 2021
ACTIVE
Proteolysis-targeting chimera against BCL-XL inhibits breast cancer metastasisNATL INST OF HLTH NCI · Co-Investigator
-
Feb 2021
–
Jan 2026
Overcoming the blood-brain barrier with nanoparticle vaccines against gliomasNATL 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 GliomasUNIV 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 tumorsST JUDE CHILDRENS RESEARCH HOSPITAL · Principal Investigator
-
Jul 2020
–
Jul 2021
Remodeling Host Immunity in Oral Cancer with Personalized RNA Nanoparticle VaccinesNATL INST OF HLTH NIDCR · Other
-
Jul 2020
–
Jun 2024
Translating Personalized RNA Nanoparticle Vaccines for Oral CancerOCALA ROYAL DAMES FOR CANCER RESEARCH · Principal Investigator
-
Jul 2020
–
Aug 2025
RNA-nanoparticle vaccines for pediatric high-grade gliomaCURESEARCH FOR CHILDRENS CANCER · Principal Investigator
-
Jun 2020
–
Nov 2023
Novel immunologic therapy of soft tissue sarcomaFL DEPT OF HLTH LIVE LIKE BELLA · Co-Investigator
-
Jun 2020
–
May 2023
Lipidnanoparticle vaccines targeting metastatic lung cancer from osteosarcomaFL 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 gliomaFL 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 TumorsDANA 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 tumorsHYUNDAI 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 GliomaICON 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 subtypesST BALDRICKS FOUNDATION · Principal Investigator
-
Jul 2019
–
Sep 2021
Immunotherapeutic targeting of osteosarcoma with nanoparticle vaccinesRally Foundation · Principal Investigator
-
Mar 2019
–
Aug 2024
CTOAUNIV 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 gliomaUF FOUNDATION · Principal Investigator
-
Jul 2018
–
Dec 2021
Legacy FoundationUF FOUNDATION · Project Manager
-
Jul 2018
–
Jun 2025
Florida Center for Brain Tumor ResearchFL 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 TumorsUNIV OF CALIFORNIA SAN FRANCISCO · Principal Investigator
-
Jan 2018
–
Jun 2020
Development of Personalized RNA Loaded Nanoparticles for Children with Refractory Solid TumorsH 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 ChildrenUNIV 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 gliomaUS ARMY MED RES ACQUISITION · Principal Investigator
-
Sep 2017
–
Jun 2021
UFHCC Triple Negative Breast Cancer (TNBC) Pilot Project ProgramMULTIPLE SPONSORS · Project Manager
-
Jul 2017
–
Dec 2019
Immunotherapeutic targeting of osteosarcoma neoantigensRally 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 MalignanciesBRISTOL MYERS SQUIBB CO · Principal Investigator
-
Apr 2017
–
Jun 2021
OoR CTSI Institutional Matching SupportUF 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 RescueNATIONWIDE CHILDRENS HOSPITAL · Principal Investigator
-
Sep 2016
ACTIVE
A Clinical and Molecular Risk-directed Therapy for Newly Diagnosed MedulloblastomaST JUDE CHILDRENS RESEARCH HOSPITAL · Principal Investigator
-
Sep 2016
–
Aug 2021
Harnessing the Immunologic Capacity of RNA-nanoparticle Vaccines Targeting GlioblastomaNATL INST OF HLTH NCI · Principal Investigator
-
Jul 2016
–
Jun 2020
RNA nanoparticle vaccines targeting malignant canine gliomasOCALA ROYAL DAMES FOR CANCER RESEARCH · Principal Investigator
-
Jul 2016
–
Jun 2020
Re-programming immunity to target mutations across medulloblastoma subtypesST BALDRICKS FOUNDATION · Principal Investigator
-
Apr 2016
–
Mar 2018
Re-programming osteosarcoma immune evasion with CAR modified T cellsUF FOUNDATION · Principal Investigator
-
Jul 2015
–
Jun 2024
Florida Academic Cancer Center Alliance Seed GrantsUF HEALTH SHANDS HOSPITAL · Project Manager
-
Dec 2014
–
Jun 2017
Efficacious, Translational and Personalized RNA-nanoparticle Vaccines Targeting Pediatric Brain TumorsHYUNDAI HOPE ON WHEELS · Principal Investigator
-
Jul 2013
–
Jun 2021
Florida Center for Brain Tumor Research – Statewide Brain Tumor Registry Program at the McKnight Brain InstituteFL DEPT OF HLTH · Project Manager
-
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
-
2013
Fellowship – Pediatric Hematology/OncologyDuke University Medical Center
-
2012-2015
Ph.D.Duke University
-
2010
Residency – PediatricsLong Island Jewish Medical Center
-
2007
M.D.University at Buffalo
-
2003
B.S.Fordham University
Ciaran Seath
- 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.
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.
- Biochemistry and Cell Biology
- Clinical and translational science
- Pediatric brain tumors
- 2025 Journal of the American Chemical Society
- 2025 bioRxiv : the preprint server for biology
- 2025 bioRxiv : the preprint server for biology
- 2025 bioRxiv : the preprint server for biology
- 2024 Trends in Biochemical Sciences
- 2024 Nano letters
- 2023 Nature reviews. Cancer
- 2022 Chemical reviews
- 2022 Journal of the American Chemical Society
- 2022 Proceedings of the National Academy of Sciences of the United States of America
- 2022 Proceedings of the National Academy of Sciences of the United States of America
- 2022 Journal of the American Chemical Society
- 2021 Chemical Society reviews
- 2019 Journal of the American Chemical Society
- 2019 Journal of the American Chemical Society
- 2019 Synlett : accounts and rapid communications in synthetic organic chemistry
-
2018
Interrogating Pd(II) Anion Metathesis Using a Bifunctional Chemical Probe: A Transmetalation Switch.Journal of the American Chemical Society
- 2018 Journal of the American Chemical Society
- 2017 Angewandte Chemie (International ed. in English)
- 2017 The Journal of organic chemistry
- 2017 Chemical communications (Cambridge, England)
- 2016 Organic letters
- 2016 Organic letters
- 2016 Chemical communications (Cambridge, England)
- 2015 Organic & biomolecular chemistry
- 2015 Chemistry (Weinheim an der Bergstrasse, Germany)
- 2015 Angewandte Chemie (International ed. in English)
- 2014 Angewandte Chemie (International ed. in English)
-
Mar 2025
ACTIVE
Nanoscale proximity labeling for chromatin chemical biologyPFIZER INC · Principal Investigator
-
Jun 2024
–
May 2025
Vanquish Neo UHPLC for The Wertheim UF Scripps InstituteNATL INST OF HLTH OD · Other
-
Jan 2024
ACTIVE
Photocatalytic Generation of Siloxycarbenes for Asymmetric OrganocatalysisAMER CHEMICAL SOC PETRO RES · Principal Investigator
-
Jul 2023
ACTIVE
Photoproximity labeling as a tool for epigenetic drug discoveryNATL INST OF HLTH NIGMS · Principal Investigator
-
May 2023
ACTIVE
Mechanisms of estrogen receptor ligand signalingNATL INST OF HLTH NCI · Co-Investigator
Maurice S Swanson
- 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.
- Aging
- Developmental regulation of RNA processing
- Genetics of Neurological diseases
- Mammalian genome repetitive elements
- Mechanisms of Regeneration
- Myotonic Dystrophy
- Neurological disorders
- RNA binding proteins
- 2025 Brain : a journal of neurology
- 2025 Nature Neuroscience
- 2025 Human Molecular Genetics
- 2025 JCI insight
- 2024 npj Microgravity
- 2024 Cell genomics
- 2024 Genes & Development
- 2024 Skeletal muscle
- 2024 bioRxiv : the preprint server for biology
- 2024 NAR Molecular Medicine
- 2024 npj Regenerative Medicine
- 2024 RNA
- 2023 Research square
- 2023 Brain : a journal of neurology
- 2023 Molecular cell
- 2022 Biology Open
- 2022 PLOS Biology
- 2021 Pathogens & immunity
- 2021 bioRxiv : the preprint server for biology
- 2020 Proceedings of the National Academy of Sciences of the United States of America
- 2020 Nature Communications
- 2020 Current biology : CB
- 2020 Genes & Development
- 2020 Nature Biomedical Engineering
- 2020 RNA
- 2019 International journal of molecular sciences
- 2019 Cerebral cortex (New York, N.Y. : 1991)
- 2019 Genes & development
- 2018 Comprehensive Physiology
- 2018 Scientific reports
- 2018 Proceedings of the National Academy of Sciences of the United States of America
- 2018 Neurobiology of disease
- 2018 Nature communications
- 2018 Nucleic acids research
- 2018 Molecular cell
- 2017 Database : the journal of biological databases and curation
- 2017 Cell reports
- 2017 Genes & development
- 2017 Cell
- 2017 Neuron
- 2017 Cell reports
- 2017 Molecular cell
- 2017 Acta neuropathologica communications
- 2016 Nature reviews. Genetics
- 2016 Molecular cell
- 2016 Nature communications
- 2016 Acta neuropathologica communications
- 2015 Proceedings of the National Academy of Sciences of the United States of America
- 2015 Nature communications
- 2015 Stem cells (Dayton, Ohio)
- 2015 Cell reports
- 2015 BMC developmental biology
- 2015 RNA biology
- 2015 Nature communications
- 2014 Brain research
- 2014 Molecular cell
- 2014 Advances in experimental medicine and biology
- 2013 Brain : a journal of neurology
- 2013 Cellular reprogramming
- 2013 Nucleic acids research
- 2013 Human molecular genetics
- 2013 Annals of neurology
- 2013 EMBO molecular medicine
- 2012 PloS one
- 2012 Neuron
- 2012 Circulation
- 2011 Proceedings of the National Academy of Sciences of the United States of America
- 2011 Cold Spring Harbor perspectives in biology
- 2011 Genetic testing and molecular biomarkers
- 2010 Nature structural & molecular biology
- 2010 Human molecular genetics
- 2010 Rinsho shinkeigaku = Clinical neurology
- 2009 The Journal of biological chemistry
- 2009 Human molecular genetics
- 2009 PLoS genetics
- 2009 Neuroscience letters
- 2007 The Journal of general physiology
- 2007 Differentiation; research in biological diversity
- 2007 Nucleic acids research
- 2007 Neuron
- 2007 Developmental biology
- 2007 The Journal of clinical investigation
- 2006 Birth defects research. Part C, Embryo today : reviews
- 2006 Human molecular genetics
- 2006 Proceedings of the National Academy of Sciences of the United States of America
- 2006 Angewandte Chemie (International ed. in English)
- 2005 The Journal of biological chemistry
- 2005 Developmental dynamics : an official publication of the American Association of Anatomists
- 2005 Journal of cell science
- 2005 Circulation research
- 2004 Nucleic acids research
- 2004 Progress in molecular and subcellular biology
- 2004 The Journal of biological chemistry
- 2004 The EMBO journal
- 2004 Journal of biomolecular NMR
- 2004 Human molecular genetics
- 2003 Gene expression patterns : GEP
- 2003 Annals of neurology
- 2003 Science (New York, N.Y.)
- 2002 The EMBO journal
- 2001 Proceedings of the National Academy of Sciences of the United States of America
- 2001 Human molecular genetics
- 2000 Genetics
- 2000 Proceedings of the National Academy of Sciences of the United States of America
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2000
Recruitment of human muscleblind proteins to (CUG)(n) expansions associated with myotonic dystrophy.The EMBO journal
- 1999 Current opinion in cell biology
- 1999 Nucleic acids research
- 1999 Methods in molecular biology (Clifton, N.J.)
- 1998 Molecular and cellular biology
- 1998 The EMBO journal
- 1997 Proceedings of the National Academy of Sciences of the United States of America
- 1996 Infection and immunity
- 1996 Cold Spring Harbor symposia on quantitative biology
- 1996 The EMBO journal
- 1996 Nucleic acids research
- 1994 JAMA
- 1994 The Journal of cell biology
- 1993 Nucleic acids research
- 1993 Molecular and cellular biology
- 1993 Molecular and cellular biology
- 1991 The EMBO journal
- 1990 Molecular biology reports
- 1990 Molecular biology reports
- 1990 Methods in enzymology
- 1989 Genes & development
- 1989 Proceedings of the National Academy of Sciences of the United States of America
- 1989 The Journal of cell biology
- 1988 Trends in biochemical sciences
- 1988 Molecular and cellular biology
- 1988 The EMBO journal
- 1987 Molecular and cellular biology
- 1986 Proceedings of the National Academy of Sciences of the United States of America
- 1986 Molecular and cellular biology
- 1985 Proceedings of the National Academy of Sciences of the United States of America
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Aug 2024
ACTIVE
Senator Paul D. Wellstone Muscular Dystrophy Specialized Research CenterNATL INST OF HLTH NINDS · Principal Investigator
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Jan 2024
ACTIVE
The molecular basis of 7SK RNA methylation in non-small cell lung cancerNATL INST OF HLTH NCI · Co-Investigator
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Sep 2023
–
Jul 2024
Mouse Dmpk CTG expansion knock in models for myotonic dystrophy (MDA 1070134)MUSCULAR DYSTROPHY ASSO · Other
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Sep 2023
ACTIVE
Understanding and targeting common mechanisms of myotonic dystrophy types 1 and 2 using novel repeat expansion mouse modelsUS ARMY MED RES ACQUISITION · Principal Investigator
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Jan 2023
–
Jun 2025
Regenerative failure in myotonic dystrophy: pathomechanisms and insights from a novel model of improved regenerationMYOTONIC DYSTROPHY FOU · Other
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Jan 2023
ACTIVE
Therapeutic strategies for microsatellite expansion diseases using RNA-targetingUNIV OF CALIFORNIA SAN DIEGO · Principal Investigator
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Aug 2021
–
Aug 2022
Mouse humanized models for myotonic dystrophy type 1 for therapy developmentKATE THERAPEUTICS · Principal Investigator
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Jul 2021
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Sep 2023
Brain Choroid Plexus Dysregulation and Cerebral Atrophy in DM1MYOTONIC DYSTROPHY FOU · Other
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Jan 2020
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Jan 2023
Provenance Initiatives Fund at the Center for NeuroGeneticsUF FOUNDATION · Co-Investigator
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Jul 2019
–
Jun 2025
RNA Processing-Mediated Mechanisms of CNS Dysfunction in Myotonic DystrophyNATL INST OF HLTH NINDS · Faculty
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Jun 2019
–
Aug 2019
International Myotonic Dystrophy Consortium Meeting IDMC-12MUSCULAR DYSTROPHY ASSO · Principal Investigator
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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 CenterUNIV OF ROCHESTER · Principal Investigator
-
Feb 2018
–
Jan 2021
Pre-mRNA Misprocessing in Myotonic Dystrophy and Amyotrophic Lateral SclerosisMUSCULAR DYSTROPHY ASSO · Other
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Jan 2018
–
Dec 2019
Congenital myotonic dystrophy:pathomechanism and therapeutic developmentWYCK FOUNDATION · Other
-
Sep 2017
–
May 2022
Therapeutic strategies for microsatellite expansion diseases using RNA-targeting CRISPR/CasUNIV OF CALIFORNIA SAN DIEGO · Principal Investigator
-
Aug 2017
–
Apr 2022
Determining the factors that control dose-dependent splicing regulation by a master regulatorNATL INST OF HLTH NIGMS · Principal Investigator
-
Feb 2017
–
Jan 2020
Novel Mouse Knockin Models for Myotonic DystrophyMUSCULAR DYSTROPHY ASSO · Principal Investigator
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Aug 2016
–
Jun 2022
Molecular Characterization of ALS/FTD in a novel C9orf72 BAC mouse model.NATL INST OF HLTH NINDS · Principal Investigator
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Feb 2016
–
Jan 2018
Myotonic Dystrophy Type 2: Mouse Models, pathomechanism and therapyWYCK FOUNDATION · Principal Investigator
-
Jan 2015
–
Jan 2018
MBI Research Program MGOLD2UF FOUNDATION · Principal Investigator
-
Sep 2013
–
Jun 2019
Myotonic Dystrophy: Molecular Pathophysiology and CNS EffectsNATL INST OF HLTH NINDS · Project Manager
-
Aug 2013
–
Jul 2016
Circadian Clock Dysregulation in Myotonic DystrophyMUSCULAR DYSTROPHY ASSO · Principal Investigator
-
Jul 2013
–
Jun 2018
Research Program MGOLD project 2UF FOUNDATION · Principal Investigator
-
Aug 2010
–
Jun 2016
RNA Dominance in Human DiseaseNATL INST OF HLTH · Principal Investigator
-
Jul 2006
–
Sep 2024
BRAIN AND SPINAL CORD INJURY RESEARCHFL DEPT OF HLTH · Project Manager
-
Issued
June 2016
Compositions and Methods Related to Protein Displacement Therapy for Myotonic Distrophy (CON)#9,371,527
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Issued
June 2011
Methods and Compositions for Treatment of Myotonia#7,964,570
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Published
March 2010
Compositions and Methods Related to Protein Displacement Therapy for Myotonic Distrophy#US-2010-0190689-A1
-
1979
PhDUniversity of California Berkeley
-
1976
MAUniversity of California Santa Barbara
Scott Tibbetts
- 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.
- 2025 Exploration of Targeted Anti-tumor Therapy
- 2024 Journal of Virology
- 2024 Journal of Virology
- 2024 Nucleic acids research
- 2024 PLOS Pathogens
- 2024 Frontiers in cellular and infection microbiology
- 2023 Journal of Virology
- 2023 mBio
- 2023 mSphere
- 2023 Trakya University Journal of Natural Sciences
- 2023 Journal of Virology
- 2022 mBio
- 2022 Proceedings of the National Academy of Sciences
- 2021 PLOS Pathogens
- 2021 Methods in molecular biology (Clifton, N.J.)
- 2021 Annual review of virology
- 2020 Scientific reports
- 2019 Non-coding RNA
- 2019 Journal of virology
- 2019 mBio
- 2019 Cell reports
- 2019 mBio
- 2019 PLOS Pathogens
- 2019 Proceedings of the National Academy of Sciences
- 2018 PLOS Pathogens
- 2018 Biochemistry
- 2018 PLoS pathogens
- 2018 Viruses
- 2018 PLoS pathogens
- 2018 Trends in cancer
- 2016 The Journal of biological chemistry
- 2016 mSphere
- 2016 Cell reports
- 2016 Journal of medical virology
- 2015 Current pathobiology reports
- 2015 mBio
- 2015 PLoS pathogens
- 2015 Nature protocols
- 2014 PLoS pathogens
- 2014 mBio
- 2014 Science (New York, N.Y.)
- 2013 Antimicrobial agents and chemotherapy
- 2012 PLoS pathogens
- 2010 PLoS pathogens
- 2010 Journal of virology
- 2010 Journal of virology
- 2008 Journal of virology
- 2006 Journal of virology
- 2006 PLoS pathogens
- 2006 Virology
- 2005 Journal of virology
- 2004 Journal of virology
- 2003 Journal of virology
- 2003 Journal of virology
- 2002 Journal of virology
- 1999 Transplantation
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Jun 2022
ACTIVE
Noncoding RNAs in gamma-Herpesvirus Biology and AIDS MalignanciesNATL INST OF HLTH NCI · Co-Investigator
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Aug 2021
ACTIVE
Gammaherpesvirus miRNA suppression of EWSR1 in GC B cell infection and lymphomagenesisNATL INST OF HLTH NCI · Principal Investigator
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Jul 2018
–
Aug 2019
The Role of a Gammaherpesvirus Noncoding RNA in Dissemination, Chronic Infection and Virus-Induced VasculitisAMER HEART ASSOCIATION · Other
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Feb 2017
–
Jan 2022
Noncoding RNAs in gamma-Herpesvirus Biology and AIDS MalignanciesNATL INST OF HLTH NCI · Faculty
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Feb 2015
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Oct 2023
UF Health Cancer Center Pilot Project Grants funded through the Florida Consortium of National Cancer Institute Centers ProgramUF HEALTH SHANDS HOSPITAL · Project Manager
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Jan 2015
–
Dec 2020
Role of MHV68 miRNAs in latency and pathogenesisNATL INST OF HLTH NIAID · Principal Investigator
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Jan 2015
–
May 2024
UF Health Cancer Center Bridge Seed Grant funded through the Florida Consortium of National Cancer Institute Centers ProgramUF HEALTH SHANDS HOSPITAL · Project Manager
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Mar 2006
–
May 2023
Davis Cancer Researach EquipmentUF FOUNDATION · Project Manager