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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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Detalhes bibliográficos
Publicado no:Nucleic Acids Res
Main Authors: Østergaard, Michael E, Jackson, Michaela, Low, Audrey, E. Chappell, Alfred, G. Lee, Richard, Peralta, Rachel Q, Yu, Jinghua, Kinberger, Garth A, Dan, Amy, Carty, Rick, Tanowitz, Michael, Anderson, Patrick, Kim, Tae-Won, Fradkin, Linda, Mullick, Adam E, Murray, Sue, Rigo, Frank, Prakash, Thazha P, Bennett, C Frank, Swayze, Eric E, Gaus, Hans J, Seth, Punit P
Formato: Artigo
Idioma:Inglês
Publicado em: Oxford University Press 2019
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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