Core Infrastructure, Capabilities and Translational Platforms
The UF RNA Genomic Medicine Center maintains a comprehensive, shared infrastructure that supports rigorous, reproducible, and scalable RNA research across UF Scripps and the University of Florida. These resources are available to Center members and the broader UF research community to accelerate discovery and translation.
Our integrated platforms enable investigators to move efficiently from molecular mechanism to therapeutic development, while ensuring high standards of quantitative validation, cross-laboratory reproducibility, and translational relevance.
RNA-Ligand Binding and BioPhysics
The Center supports quantitative analysis of RNA–small molecule and RNA–protein interactions using state-of-the-art biophysical instrumentation, including:
- Microscale Thermophoresis (MST) for affinity and binding kinetics
- Differential Scanning Calorimetry (DSC) for thermal stability and conformational analysis
- NanoTemper Prometheus systems for thermal unfolding and binding characterization
- Isothermal Titration Calorimetry (ITC) for thermodynamic profiling
- Surface Plasmon Resonance (SPR) for real-time binding and kinetic measurements
These platforms enable rigorous benchmarking of ligand engagement, selectivity, and mechanism of action.
RNA-Targeted Small Molecule Discovery
The Center provides specialized resources for RNA-focused chemical biology and drug discovery, including:
- Curated RNA-focused small molecule and fragment libraries
- Covalent and proximity-based screening platforms
Structural Biology and Molecular Characterization
The Center integrates complementary structural approaches for RNA and RNA–ligand complexes, including:
- Cryo-Electron Microscopy (Cryo-EM)
- Nuclear Magnetic Resonance (NMR) spectroscopy
- X-ray crystallography
- Biophysical and computational modeling
This multi-platform approach enables atomic- and near-atomic-resolution characterization of RNA structure, dynamics, and ligand binding.
RNA Structure, Mapping and Informatics
The Center operates advanced platforms for defining RNA structure–function relationships, including:
- Chemical probing technologies for RNA structure mapping
- Evolutionary covariance analysis
- AI-enabled and computational modeling tools
- Integrated informatics pipelines for structure–function annotation
These capabilities support systematic analysis of RNA folding, conformational dynamics, and interaction networks.
Long-Read and Nanopore Sequencing Technologies
The Center maintains dedicated infrastructure for comprehensive long-read RNA analysis, including:
- Nanopore sequencing platforms
- Long-read chemical probing workflows
- Isoform-resolved transcriptome analysis
- RNA modification and processing analysis
- Single-molecule RNA profiling
These technologies enable detailed characterization of RNA isoforms, splicing patterns, structural states, and regulatory features that cannot be resolved using short-read methods.
Integrated Genomics, Proteomics and Systems Biology Cores
In addition to center-specific platforms, investigators have access to:
- Wertheim UF Scripps Proteomics Core
- UF Genomics and Sequencing Cores
- Mass spectrometry facilities
- High-performance computing and data storage infrastructure
- Bioinformatics and statistical support
These shared resources support multi-omic and systems-level studies.
RNA-to-Therapeutic Evaluation and Degrader Development
A defining core competency of the UF RNA Genomic Medicine Center is the ability to evaluate RNA targets and determine their therapeutic tractability.
For any RNA target of interest, the Center provides integrated capabilities to:
- Assess structural features, accessibility, and regulatory context
- Analyze sequence conservation and evolutionary constraints
- Map functional RNA motifs and interaction networks
- Evaluate druggability using experimental and computational platforms
These analyses enable systematic assessment of the probability that a given RNA can be modulated by small molecules.
When targets are deemed tractable, the Center supports development of multiple classes of RNA-targeted agents, including:
Binding and Functional Modulators
Small molecules that selectively bind RNA to modulate splicing, translation, localization, or stability.
Direct Chemical Degraders
Compounds designed to induce oxidative or chemical cleavage of RNA through intrinsic reactivity or proximity-enabled chemistry.
Enzyme-Recruiting Degraders
Molecules that recruit endogenous effector proteins and nucleases to promote selective RNA degradation through proximity-induced mechanisms.
Together, these approaches enable both occupancy-based and event-driven strategies for RNA control.
Enabling a Gene → RNA →Drug Paradigm
These integrated capabilities support a unified gene → RNA → drug paradigm for precision medicine.
The Center has applied this framework broadly across:
- Coding RNAs
- Long noncoding RNAs
- Repeat expansion RNAs
- Viral RNAs
- Regulatory and structural RNAs
This approach enables rapid progression from genetic association and mechanistic insight to therapeutic intervention within a single coordinated ecosystem.
Translational, In Vivo and Platform Development Expertise
Beyond instrumentation, the Center provides deep technical and scientific expertise in translating RNA discoveries into validated therapeutic candidates.
For many RNA-mediated diseases—particularly in oncology and repeat expansion disorders—the Center maintains extensive experience in developing and applying relevant cellular and animal models. These capabilities enable systematic evaluation of RNA-modulating compounds from biochemical and biophysical assays through cellular systems and into in vivo disease models.
Core translational competencies include:
- RNA-targeted therapeutic design across multiple modalities
- Chemical biology and covalent ligand development
- AI-guided discovery and optimization
- Preclinical assay development and validation
- Mechanism-of-action and target engagement studies
- Biomarker and pharmacodynamic analysis
- Generation and characterization of disease-relevant mouse models
- In vivo efficacy, toxicity, and pharmacokinetic studies
By coupling molecular characterization with robust in vivo validation, the Center ensures that therapeutic candidates entering translational pipelines are mechanistically grounded, biologically validated, and positioned for clinical advancement.
Access, Outreach and Community Engagement
Center infrastructure is available to:
- Wertheim UF Scripps and UF Gainesville investigators
- External academic collaborators
- Industry partners
- Translational and commercialization programs
Access is supported through shared facilities, collaborative agreements, and project-based partnerships.
The Center actively promotes resource sharing, standardized workflows, and cross-laboratory validation to ensure high-quality, reproducible research.
We are committed to evolving our infrastructure in response to community needs. Investigators and collaborators are encouraged to provide feedback on emerging opportunities and areas for enhancement. Input can be directed to Center and Institute leadership.
Enabling Impact
By integrating advanced instrumentation, computational platforms, translational expertise, and in vivo validation capabilities, the UF RNA Genomic Medicine Center provides a unified environment for RNA discovery and therapeutic innovation.
These shared resources shorten the path from fundamental insight to clinical and commercial impact, while strengthening Florida’s position as a global leader in RNA science.
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