ArticleProceedings of the National Academy of Sciences of the United States of America2019
Intrinsically cell-penetrating multivalent and multitargeting ligands for myotonic dystrophy type 1.
Article in Proceedings of the National Academy of Sciences of the United States of America, 2019. The graph could read no effect estimate from its abstract, so it casts no vote on the map. Cited by 33 papers.
What it found
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Who cites it
33 citing papers in PubMed, 48 citations in OpenAlex.
- RNA-binding landscape of amiloride: large-scale profiling and structural basis of U-U mismatch recognition.RSC chemical biology · 2026Article
- Targeting Expanded CUG and CTG Repeats as a Therapeutic Approach for Myotonic Dystrophy Type 1 (DM1).ChemMedChem · 2026Review
- Article
- SynBioNanoDesign: pioneering targeted drug delivery with engineered nanomaterials.Journal of nanobiotechnology · 2025Review
- Altered drug metabolism and increased susceptibility to fatty liver disease in a mouse model of myotonic dystrophy.Nature communications · 2024Article
- Large-scale analysis of small molecule-RNA interactions using multiplexed RNA structure libraries.Communications chemistry · 2024Article
- Small molecules modulating RNA splicing: a review of targets and future perspectives.RSC medicinal chemistry · 2024Review
- High-Throughput Synthesis, Purification, and Application of Alkyne-Functionalized Discrete Oligomers.Journal of the American Chemical Society · 2024Article
- Multivalent ACE2 engineering-A promising pathway for advanced coronavirus nanomedicine development.Nano today · 2022Review
- NMR determination of the 2:1 binding complex of naphthyridine carbamate dimer (NCD) and CGG/CGG triad in double-stranded DNA.Nucleic acids research · 2022Article
- Development of Therapeutic Approaches for Myotonic Dystrophies Type 1 and Type 2.International journal of molecular sciences · 2022Review
- Selective and Reversible Ligand Assembly on the DNA and RNA Repeat Sequences in Myotonic Dystrophy.Chembiochem : a European journal of chemical biology · 2022Article
- Molecular Therapies for Myotonic Dystrophy Type 1: From Small Drugs to Gene Editing.International journal of molecular sciences · 2022Review
- Disrupting the Molecular Pathway in Myotonic Dystrophy.International journal of molecular sciences · 2021Review
- A Novel Minor Groove Binder as a Potential Therapeutic Agent for Myotonic Dystrophy Type 1.ChemMedChem · 2021Article
- Versatile Target-Guided Screen for Discovering Bidirectional Transcription Inhibitors of a Trinucleotide Repeat Disease.ACS medicinal chemistry letters · 2021Article
- Affecting RNA biology genome-wide by binding small molecules and chemically induced proximity.Current opinion in chemical biology · 2021Review
- A polymeric approach toward resistance-resistant antimicrobial agent with dual-selective mechanisms of action.Science advances · 2021Article
- Design of novel small molecule base-pair recognizers of toxic CUG RNA transcripts characteristics of DM1.Computational and structural biotechnology journal · 2021Article
- Small molecule-RNA targeting: starting with the fundamentals.Chemical communications (Cambridge, England) · 2020Article
Corrections and comments
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Authors and funding
11 authors at 3 institutions in 3 countries.
Funding
Abstract
Developing highly active, multivalent ligands as therapeutic agents is challenging because of delivery issues, limited cell permeability, and toxicity. Here, we report intrinsically cell-penetrating multivalent ligands that target the trinucleotide repeat DNA and RNA in myotonic dystrophy type 1 (DM1), interrupting the disease progression in two ways. The oligomeric ligands are designed based on the repetitive structure of the target with recognition moieties alternating with bisamidinium groove binders to provide an amphiphilic and polycationic structure, mimicking cell-penetrating peptides. Multiple biological studies suggested the success of our multivalency strategy. The designed oligomers maintained cell permeability and exhibited no apparent toxicity both in cells and in mice at working concentrations. Furthermore, the oligomers showed important activities in DM1 cells and in a DM1 liver mouse model, reducing or eliminating prominent DM1 features. Phenotypic recovery of the climbing defect in adult DM1
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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.