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Estrogen‐Induced LncRNA, LINC02568, Promotes Estrogen Receptor‐Positive Breast Cancer Development and Drug Resistance Through Both InTrans and In Cis Mechanisms

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Targeting RNA with small molecules, from RNA structures to precision medicines: IUPHAR review: 40

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A blood–brain penetrant RNA-targeted small molecule triggers elimination of r(G 4 C 2 ) exp in c9ALS/FTD via the nuclear RNA exosome

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Targeted Degradation of Structured RNAs via Ribonuclease-Targeting Chimeras (RiboTacs)

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CAG-Repeat RNA Hairpin Folding and Recruitment to Nuclear Speckles with a Pivotal Role of ATP as a Cosolute

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Transcriptome-Wide, Unbiased Profiling of Ribonuclease Targeting Chimeras

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Small Molecule RNA Degraders

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Synthesis and evaluation of RNase L-binding 2-aminothiophenes as anticancer agents

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Exploring the therapeutic potential of modulating nonsense-mediated mRNA decay

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Discovery of RNA‐Targeting Small Molecules: Challenges and Future Directions

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Kaempferol enhances ER-mitochondria coupling and protects motor neurons from mitochondrial dysfunction and ER stress in C9ORF72-ALS

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Targeting Ribonucleases with Small Molecules and Bifunctional Molecules

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Targeting neurodegenerative disease-associated protein aggregation with proximity-inducing modalities

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Deciphering the regulatory landscape of enhancer RNAs in health and disease

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The Druggable Transcriptome Project: From Chemical Probes to Precision Medicines

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Mapping small molecule–RNA binding sites via Chem-CLIP synergized with capillary electrophoresis and nanopore sequencing

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Development of Nitroso-Based Probes for Labeling and Regulation of RAS Proteins in Cancer Cells via Sequential Ene-Ligation and Oxime Condensation

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Heterobifunctional small molecules to modulate RNA function

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High-throughput screen of 100 000 small molecules in C9ORF72 ALS neurons identifies spliceosome modulators that mobilize G4C2 repeat RNA into nuclear export and repeat associated non-canonical translation

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Sequential Azidation/Azolation of Prenylated Derivatives and a Click Reaction Enable Selective Labeling and Degradation of RAS Protein

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RNA G‐Quadruplex RIBOTAC‐Mediated Targeted Degradation of lncRNA TERRA

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Major Advances in Emerging Degrader Technologies

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Therapeutic Approaches for C9ORF72-Related ALS: Current Strategies and Future Horizons

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Targeting RNA binding proteins with small-molecule inhibitors: Advances, challenges, and therapeutic opportunities

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High-Resolution NMR Structures of Intrastrand Hairpins Formed by CTG Trinucleotide Repeats

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Aspects of degradation and translation of the expanded C9orf72 hexanucleotide repeat RNA

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Drugging “undruggable” neurodegenerative disease targets with small molecules

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Fueling the revolution: RIBOTACs manipulating RNA decay

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Targeting Pathogenic DNA and RNA Repeats: A Conceptual Therapeutic Way for Repeat Expansion Diseases

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Catalytic Protein Inhibitors

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Dynamic docking of small molecules targeting RNA CUG repeats causing myotonic dystrophy type 1

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PROTACs for central nervous system disorders: challenges and opportunities

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Multifaceted targeted protein degradation systems for different cellular compartments

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Design of an Orally Bioavailable Small Molecule That Modulates the Microtubule-Associated Protein Tau’s Pre-mRNA Splicing

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A Live-Cell NanoBRET Assay to Monitor RNA–Protein Interactions and Their Inhibition by Small Molecules

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Engineered GM1 Intersects Between Mitochondrial and Synaptic Pathways to Ameliorate ALS Pathology

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Degrading disease-causing RNA in ALS

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Small Molecules Targeting Repeat Sequences Causing Neurological Disorders

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RNAs in the diagnosis and treatment of major diseases

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Experimental and computational investigations of RNA duplexes containing N7-regioisomers of adenosine and LNA-adenosine

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Screening and Lead Generation Techniques for RNA Binders

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Small Molecules Targeting Repeat Sequences Causing Neurological Disorders

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Extending the Degradation Toolkit – mRNA Targeting as an Alternative Means to Affect Protein Levels

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The Future of Heterobifunctional Compounds: PROTACs and Beyond

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Catalytic Protein Inhibitors

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RNP granules in ALS and neurodegeneration: From multifunctional membraneless organelles to therapeutic opportunities

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Small Molecules That Degrade RNA

Springer, N. A., Meyer, S. M., Taghavi, A., Childs‐Disney, J. L., & Disney, M. D. (2024) · Methods and principles in medicinal chemistry · doi:10.1002/9783527840458.ch9

Small Molecule RNA Degraders

Bonet‐Aleta, J., Maehara, T., Craig, B. A., & Bernardes, G. J. L. (2024) · Angewandte Chemie · doi:10.1002/ange.202412925

RNA modulation

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RNA-Focused DNA-Encoded Library Construction, Screening, and Integration of Docking Identify Bioactive Ligands of Pathogenic r(G 4 C 2 ) exp RNAPreprint

Yang, X., Taghavi, A., Akahori, Y., Pedrini, M., Ishii, T. M., & Disney, M. D. (2025) · bioRxiv (Cold Spring Harbor Laboratory) · doi:10.1101/2025.11.06.686979

Opportunities and challenges in targeted protein degradation for antiviral drug discovery

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An RNA-Focused DNA-Encoded Library Platform for Discovering Ligands of Pathogenic r(G 4 C 2 ) exp RNA

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Small-molecule-mediated RNA targeting: emerging strategies and therapeutic potential of RiboTAC

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RIBOTAC-mediated degradation of hsa-microRNA-301a-3p suppresses TNBC bone metastasis

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BindingDB Entry 50016515: Nucleic-Acid-Based Targeted Degradation in Drug Discovery.

BindingDB (2023) · UC San Diego · doi:10.7270/q2k35zpc

Degrading disease-causing RNA in ALS

Cully, M. (2021) · Nature Reviews Drug Discovery · doi:10.1038/d41573-021-00189-2 · Cited by 1 · see citing works

RNA-seq of a small molecule targeted recruitment of a ribonuclease to degrade the c9ALS/FTD r(G4C2) repeat expansion

Tong, Y. (2021) · Data Archiving and Networked Services (DANS) · doi:10.17632/332kvzz8xk

A blood–brain penetrant RNA-targeted small molecule triggers elimination of r(G 4 C 2 ) exp in c9ALS/FTD via the nuclear RNA exosome

Bush, J. A., Meyer, S. M., Fuerst, R., Tong, Y., Li, Y., Benhamou, R. I., Aikawa, H., Zanon, P. R. A., Gibaut, Q. M. R., Angelbello, A. J., et al. (2022) · Proceedings of the National Academy of Sciences · doi:10.1073/pnas.2210532119 · Cited by 23 · see citing works

RNA-seq data for a small molecule alleviating C9orf72 ALS pathology by interfacing G4C2 RNA repeats with endogenous decay pathways

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A Small Molecule Exploits Hidden Structural Features within the RNA Repeat Expansion That Causes c9ALS/FTD and Rescues Pathological Hallmarks

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