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- W2938190307 abstract "Significance Multivalent ligands have tremendous potential as therapeutic agents; however, their efficacy is limited by delivery issues, poor cell permeability, and toxicity. We report here a strategy wherein multivalent ligands are designed to be intrinsically cell-penetrating, allowing them to target the expanded trinucleotide repeat sequences of DNA and RNA that cause myotonic dystrophy type 1 (DM1). The multivalent ligand studied shows cell permeability and low toxicity both in cells and in mice. Importantly, the ligand reduced or eliminated DM1 defects in DM1 cells and in vivo, validating the multivalent strategy. The approach should be broadly applicable to other repeat expansion diseases and to any multivalent oligomeric therapeutic agent whose activity can accommodate structural elements that mimic cell-penetrating peptides." @default.
- W2938190307 created "2019-04-25" @default.
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- W2938190307 date "2019-04-11" @default.
- W2938190307 modified "2023-09-26" @default.
- W2938190307 title "Intrinsically cell-penetrating multivalent and multitargeting ligands for myotonic dystrophy type 1" @default.
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- W2938190307 doi "https://doi.org/10.1073/pnas.1820827116" @default.
- W2938190307 hasPubMedCentralId "https://www.ncbi.nlm.nih.gov/pmc/articles/6500145" @default.
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