ArticleNucleic acids research2013
Single-molecule study of the CUG repeat-MBNL1 interaction and its inhibition by small molecules.
Article in Nucleic acids research, 2013. The graph could read no effect estimate from its abstract, so it casts no vote on the map. Cited by 21 papers.
What it found
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The abstract states no effect estimate the extractor could read, or names no intervention and outcome on the map, so this paper lights no cell and moves no belief. It is still indexed, cited and linked below.
The trial behind it
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Who cites it
21 citing papers in PubMed, 34 citations in OpenAlex.
- 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
- A pothole-filling strategy for selective targeting of rCUG-repeats associated with myotonic dystrophy type 1.Proceedings of the National Academy of Sciences of the United States of America · 2026Article
- Transcriptome-Wide Studies of RNA-Targeted Small Molecules Provide a Simple and Selective r(CUG)ACS central science · 2023Article
- Article
- Possibilities and challenges of small molecule organic compounds for the treatment of repeat diseases.Proceedings of the Japan Academy. Series B, Physical and biological sciences · 2022Article
- The powerful world of antisense oligonucleotides: From bench to bedside.Wiley interdisciplinary reviews. RNA · 2020Review
- Quantitative Studies of Muscleblind Proteins and Their Interaction With TCF4 RNA Foci Support Involvement in the Mechanism of Fuchs' Dystrophy.Investigative ophthalmology & visual science · 2019Article
- Myotonic Dystrophy and Developmental Regulation of RNA Processing.Comprehensive Physiology · 2018Review
- Myotonic dystrophy: approach to therapy.Current opinion in genetics & development · 2017Review
- PCNA tool belts and polymerase bridges form during translesion synthesis.Nucleic acids research · 2016Article
- A Potent Inhibitor of Protein Sequestration by Expanded Triplet (CUG) Repeats that Shows Phenotypic Improvements in a Drosophila Model of Myotonic Dystrophy.ChemMedChem · 2016Article
- The Proliferating Cell Nuclear Antigen (PCNA)-interacting Protein (PIP) Motif of DNA Polymerase η Mediates Its Interaction with the C-terminal Domain of Rev1.The Journal of biological chemistry · 2016Article
- A journey in bioinspired supramolecular chemistry: from molecular tweezers to small molecules that target myotonic dystrophy.Beilstein journal of organic chemistry · 2016Review
- Quantifying the Assembly of Multicomponent Molecular Machines by Single-Molecule Total Internal Reflection Fluorescence Microscopy.Methods in enzymology · 2016Article
- Direct correlation of DNA binding and single protein domain motion via dual illumination fluorescence microscopy.Nano letters · 2014Article
- Lomofungin and dilomofungin: inhibitors of MBNL1-CUG RNA binding with distinct cellular effects.Nucleic acids research · 2014Article
- Mismatch repair protein hMSH2-hMSH6 recognizes mismatches and forms sliding clamps within a D-loop recombination intermediate.Proceedings of the National Academy of Sciences of the United States of America · 2014Article
- Small molecule kinase inhibitors alleviate different molecular features of myotonic dystrophy type 1.RNA biology · 2014Article
- MBNL proteins and their target RNAs, interaction and splicing regulation.Nucleic acids research · 2014Review
Corrections and comments
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Authors and funding
4 authors at 2 institutions in 1 country.
Funding
Abstract
Effective drug discovery and optimization can be accelerated by techniques capable of deconvoluting the complexities often present in targeted biological systems. We report a single-molecule approach to study the binding of an alternative splicing regulator, muscleblind-like 1 protein (MBNL1), to (CUG)n = 4,6 and the effect of small molecules on this interaction. Expanded CUG repeats (CUG(exp)) are the causative agent of myotonic dystrophy type 1 by sequestering MBNL1. MBNL1 is able to bind to the (CUG)n-inhibitor complex, indicating that the inhibition is not a straightforward competitive process. A simple ligand, highly selective for CUG(exp), was used to design a new dimeric ligand that binds to (CUG)n almost 50-fold more tightly and is more effective in destabilizing MBNL1-(CUG)4. The single-molecule method and the analysis framework might be extended to the study of other biomolecular interactions.
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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.