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Intrinsically cell-penetrating multivalent and multitargeting ligands for myotonic dystrophy type 1

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 (DM...

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Bibliografiske detaljer
Udgivet i:Proc Natl Acad Sci U S A
Main Authors: Lee, JuYeon, Bai, Yugang, Chembazhi, Ullas V., Peng, Shaohong, Yum, Kevin, Luu, Long M., Hagler, Lauren D., Serrano, Julio F., Chan, H. Y. Edwin, Kalsotra, Auinash, Zimmerman, Steven C.
Format: Artigo
Sprog:Inglês
Udgivet: National Academy of Sciences 2019
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Online adgang:https://ncbi.nlm.nih.gov/pmc/articles/PMC6500145/
https://ncbi.nlm.nih.gov/pubmed/30975744
https://ncbi.nlm.nih.govhttp://dx.doi.org/10.1073/pnas.1820827116
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