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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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Detalles Bibliográficos
Autores principales:	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:	Online Artículo Texto
Lenguaje:	English
Publicado:	Oxford University Press 2019
Materias:	Chemical Biology and Nucleic Acid Chemistry
Acceso en línea:	https://www.ncbi.nlm.nih.gov/pmc/articles/PMC6614849/ https://www.ncbi.nlm.nih.gov/pubmed/31076766 http://dx.doi.org/10.1093/nar/gkz360

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