ArticleJournal of medicinal chemistry2013
Developing bivalent ligands to target CUG triplet repeats, the causative agent of myotonic dystrophy type 1.
Article in Journal of medicinal chemistry, 2013. The graph could read no effect estimate from its abstract, so it casts no vote on the map. Cited by 33 papers.
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The trial behind it
Trials whose registry record cites this paper, or whose number appears in the abstract. A trial that started after this paper was published is citing it as background, not reporting it.
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Who cites it
33 citing papers in PubMed.
- Modified Polycyclic Compounds Rescue Mis-splicing in Myotonic Dystrophy Type 1 Disease Models.ACS chemical biology · 2026Article
- Targeting Expanded CUG and CTG Repeats as a Therapeutic Approach for Myotonic Dystrophy Type 1 (DM1).ChemMedChem · 2026Review
- Targeting RNA structures with small molecules.Nature reviews. Drug discovery · 2022Review
- A Novel Minor Groove Binder as a Potential Therapeutic Agent for Myotonic Dystrophy Type 1.ChemMedChem · 2021Article
- A Small Molecule that Binds an RNA Repeat Expansion Stimulates Its Decay via the Exosome Complex.Cell chemical biology · 2021Article
- Design of small molecules targeting RNA structure from sequence.Chemical Society reviews · 2020Review
- Small molecule recognition of disease-relevant RNA structures.Chemical Society reviews · 2020Review
- Expanded DNA and RNA Trinucleotide Repeats in Myotonic Dystrophy Type 1 Select Their Own Multitarget, Sequence-Selective Inhibitors.Biochemistry · 2020Article
- Review
- S-phase Enriched Non-coding RNAs Regulate Gene Expression and Cell Cycle Progression.Cell reports · 2020Article
- Article
- Development of novel macrocyclic small molecules that target CTG trinucleotide repeats.Bioorganic & medicinal chemistry · 2019Article
- Unveiling the druggable RNA targets and small molecule therapeutics.Bioorganic & medicinal chemistry · 2019Review
- Pharmaceutical Applications of Molecular Tweezers, Clefts and Clips.Molecules (Basel, Switzerland) · 2019Review
- Intrinsically cell-penetrating multivalent and multitargeting ligands for myotonic dystrophy type 1.Proceedings of the National Academy of Sciences of the United States of America · 2019Article
- The TwistDock workflow for evaluation of bivalent Smac mimetics targeting XIAP.Drug design, development and therapy · 2019Article
- Drugging the RNA World.Cold Spring Harbor perspectives in biology · 2018Review
- Targeting RNA in mammalian systems with small molecules.Wiley interdisciplinary reviews. RNA · 2018Review
- Myotonic dystrophy: approach to therapy.Current opinion in genetics & development · 2017Review
- Modeling ALS and FTD with iPSC-derived neurons.Brain research · 2017Review
Corrections and comments
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Authors and funding
7 authors.
Funding
Abstract
An expanded CUG repeat transcript (CUG(exp)) is the causative agent of myotonic dystrophy type 1 (DM1) by sequestering muscleblind-like 1 protein (MBNL1), a regulator of alternative splicing. On the basis of a ligand (1) that was previously reported to be active in an in vitro assay, we present the synthesis of a small library containing 10 dimeric ligands (4-13) that differ in length, composition, and attachment point of the linking chain. The oligoamino linkers gave a greater gain in affinity for CUG RNA and were more effective when compared to oligoether linkers. The most potent in vitro ligand (9) was shown to be aqueous-soluble and both cell- and nucleus-permeable, displaying almost complete dispersion of MBNL1 ribonuclear foci in a DM1 cell model. Direct evidence for the bioactivity of 9 was observed in its ability to disperse ribonuclear foci in individual live DM1 model cells using time-lapse confocal fluorescence microscopy.
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Registered trials
Read under generation 80e0d062 · epoch 390. Bibliography from PubMed, PubMed Central and OpenAlex; grants from NIH RePORTER; trial links from ClinicalTrials.gov; estimates, votes and beliefs from the OpenQuestion graph.