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Conjugation of hydrophobic moieties enhances potency of antisense oligonucleotides in the muscle of rodents and non-human primates
We determined the effect of attaching palmitate, tocopherol or cholesterol to PS ASOs and their effects on plasma protein binding and on enhancing ASO potency in the muscle of rodents and monkeys. We found that cholesterol ASO conjugates showed 5-fold potency enhancement in the muscle of rodents rel...
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Publicado no: | Nucleic Acids Res |
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Main Authors: | , , , , , , , , , , , , , , , , , , , , , |
Formato: | Artigo |
Idioma: | Inglês |
Publicado em: |
Oxford University Press
2019
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Assuntos: | |
Acesso em linha: | https://ncbi.nlm.nih.gov/pmc/articles/PMC6614849/ https://ncbi.nlm.nih.gov/pubmed/31076766 https://ncbi.nlm.nih.govhttp://dx.doi.org/10.1093/nar/gkz360 |
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