ArticleCell chemical biology2021
A Small Molecule that Binds an RNA Repeat Expansion Stimulates Its Decay via the Exosome Complex.
Open access · bronzeAbstract read
In one paragraph
Article in Cell chemical biology, 2021. The graph could read no effect estimate from its abstract, so it casts no vote on the map. Cited by 28 papers.
0numbers the graph read from it
0cells of the map it votes in
28citing papers in PubMed
1.9field-weighted citation impact, top 13% of its field
What it found
Each row is one number read from the abstract, on the scale the paper reported it, with its interval. Left of the dashed line favours the treatment, right favours the comparator. Under each row is the sentence it came from. New to these charts? A ten-minute tutorial.
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
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.
Neither the registry nor the abstract names a trial number. If this is a trial report, that itself is worth knowing.
Who cites it
28 citing papers in PubMed, 45 citations in OpenAlex.
- Beyond Coding Variants: RNA-Level Mechanisms in Human Disease and Precision Therapeutics.Genes · 2026Review
- Exploring the histopathological signature of repeat-mediated Fuchs endothelial corneal dystrophy.Acta ophthalmologica · 2026Article
- Comparative analysis of alkyne- and desthiobiotinylated photoaffinity probes for chemotranscriptomic profiling.RSC chemical biology · 2026Article
- 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
- Predicting small molecule-RNA interactions without RNA tertiary structures.Nature biotechnology · 2026Article
- Unbiased RNA Degrader Identification Uncovers an LC3B-Recruiting Chimera forJournal of the American Chemical Society · 2025Article
- Docking and Molecular Dynamics Simulation-Based Analysis of Advanced Small-Molecule Kinase Inhibitors Identified pre-let-7 miRNA Binders.Chembiochem : a European journal of chemical biology · 2025Article
- The Druggable Transcriptome Project: From Chemical Probes to Precision Medicines.Biochemistry · 2025Review
- How "Omics" Studies Contribute to a Better Understanding of Fuchs' Endothelial Corneal Dystrophy.Current issues in molecular biology · 2025Review
- Experimental and computational investigations of RNA duplexes containing N7-regioisomers of adenosine and LNA-adenosine.Nucleic acids research · 2025Article
- Targeting RNA with small molecules, from RNA structures to precision medicines: IUPHAR review: 40.British journal of pharmacology · 2024Review
- Tissue-specific TCF4 triplet repeat instability revealed by optical genome mapping.EBioMedicine · 2024Article
- Therapeutic targeting of RNA for neurological and neuromuscular disease.Genes & development · 2024Review
- Transcriptome-Wide, Unbiased Profiling of Ribonuclease Targeting Chimeras.Journal of the American Chemical Society · 2024Article
- Deciphering novel TCF4-driven mechanisms underlying a common triplet repeat expansion-mediated disease.PLoS genetics · 2024Article
- Transcriptome-Wide Studies of RNA-Targeted Small Molecules Provide a Simple and Selective r(CUG)ACS central science · 2023Article
- Loss of Corneal Nerves and Corneal Haze in Patients with Fuchs' Endothelial Corneal Dystrophy with the Transcription Factor 4 Gene Trinucleotide Repeat Expansion.Ophthalmology science · 2023Article
- Dynamic docking of small molecules targeting RNA CUG repeats causing myotonic dystrophy type 1.Biophysical journal · 2023Article
- A blood-brain penetrant RNA-targeted small molecule triggers elimination of r(GProceedings of the National Academy of Sciences of the United States of America · 2022Article
Corrections and comments
- Commented on by
Authors and funding
12 authors at 4 institutions in 1 country.
Alicia J AngelbelloDepartment of Chemistry, The Scripps Research Institute, Jupiter, FL 33458, USA.
Raphael I BenhamouDepartment of Chemistry, The Scripps Research Institute, Jupiter, FL 33458, USA.
Suzanne G RzuczekDepartment of Chemistry, The Scripps Research Institute, Jupiter, FL 33458, USA.
Shruti ChoudharyDepartment of Chemistry, The Scripps Research Institute, Jupiter, FL 33458, USA.
Zhenzhi TangDepartment of Neurology, School of Medicine and Dentistry, University of Rochester, Rochester, NY 14642, USA.
Jonathan L ChenDepartment of Chemistry, The Scripps Research Institute, Jupiter, FL 33458, USA.
Madhuparna RoyWilmer Eye Institute, Johns Hopkins Medical Institutions, Baltimore, MD 21287, USA.
Kye Won WangDepartment of Chemistry, Florida Atlantic University, Jupiter, FL 33458, USA.
Ilyas YildirimDepartment of Chemistry, Florida Atlantic University, Jupiter, FL 33458, USA.
Albert S JunWilmer Eye Institute, Johns Hopkins Medical Institutions, Baltimore, MD 21287, USA.
Charles A ThorntonDepartment of Neurology, School of Medicine and Dentistry, University of Rochester, Rochester, NY 14642, USA.
Matthew D DisneyDepartment of Chemistry, The Scripps Research Institute, Jupiter, FL 33458, USA. Electronic address: disney@scripps.edu.
Scripps Research Institute · USFlorida Atlantic University · USJohns Hopkins University · USUniversity of Rochester · US
Funding
Design of Precision Small Molecules Targeting RNA Repeating Transcripts to Manipulate and Study Disease BiologyR35NS116846 · NINDS · UNIVERSITY OF FLORIDA · PI Matthew D Disney · 2020 to 2026
$9.4MSenator Paul D. Wellstone Muscular Dystrophy Specialized Research CenterP50NS048843 · NINDS · UNIVERSITY OF ROCHESTER · PI THORNTON, CHARLES A · 2018 to 2022
$7.1MUsing a disease-affected cell to synthesize its own drugDP1NS096898 · NINDS · SCRIPPS FLORIDA · PI DISNEY, MATTHEW D · 2015 to 2019
$5.0MTherapeutic Modulation of Myotonic Muscular DystrophyR37NS094393 · NINDS · UNIVERSITY OF ROCHESTER · PI THORNTON, CHARLES A · 2015 to 2021
$2.5MAcquisition of a Typhoon FLA 9500 Laser Scanning ImagerS10OD021489 · OD · UNIVERSITY OF ROCHESTER · PI BUTLER, JAMES SCOTT · 2017 to 2017
$130kChemical approaches for precise recognition of a toxic RNA repeatF31NS110269 · NINDS · SCRIPPS FLORIDA · PI ANGELBELLO, ALICIA JEANNINE · 2018 to 2020
$64kNIH HHS S10 OD021489NINDS NIH HHS DP1 NS096898NINDS NIH HHS F31 NS110269NINDS NIH HHS P50 NS048843NINDS NIH HHS R35 NS116846NINDS NIH HHS R37 NS094393
Abstract
Many diseases are caused by toxic RNA repeats. Herein, we designed a lead small molecule that binds the structure of the r(CUG) repeat expansion [r(CUG)
Indexed as
Cells, CulturedExosomesFemaleFuchs' Endothelial DystrophyHumansMaleMyotonic DystrophySmall Molecule LibrariesTrinucleotide Repeat ExpansionSmall Molecule Librarieschemical biologydecay pathwaysdrug discoverymicrosatellite disordersRNARNA splicingtargeted degradation
Identifiers
What OpenQuestion holds
Textmetadata
LicenceTDM
Read underepoch 390
Registered trials
None linked
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.