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A Novel Family of Small Molecules that Enhance the Intracellular Delivery and Pharmacological Effectiveness of Antisense and Splice Switching Oligonucleotides

The pharmacological effectiveness of oligonucleotides has been hampered by their tendency to remain entrapped in endosomes thus limiting their access to cytosolic or nuclear targets. We have previously reported a group of small molecules that enhance the effects of oligonucleotides by causing their...

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Veröffentlicht in:ACS Chem Biol
Hauptverfasser: Wang, Ling, Ariyarathna, Yamuna, Ming, Xin, Yang, Bing, James, Lindsey I., Kreda, Silvia M., Porter, Melissa, Janzen, William, Juliano, Rudolph L.
Format: Artigo
Sprache:Inglês
Veröffentlicht: 2017
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Online Zugang:https://ncbi.nlm.nih.gov/pmc/articles/PMC5662188/
https://ncbi.nlm.nih.gov/pubmed/28703575
https://ncbi.nlm.nih.govhttp://dx.doi.org/10.1021/acschembio.7b00242
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