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Asialoglycoprotein receptor 1 mediates productive uptake of N-acetylgalactosamine-conjugated and unconjugated phosphorothioate antisense oligonucleotides into liver hepatocytes

Antisense oligonucleotide (ASO) therapeutics show tremendous promise for the treatment of previously intractable human diseases but to exert their effects on cellular RNA processing they must first cross the plasma membrane by endocytosis. The conjugation of ASOs to a receptor ligand can dramaticall...

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Detalhes bibliográficos
Publicado no:Nucleic Acids Res
Main Authors: Tanowitz, Michael, Hettrick, Lisa, Revenko, Alexey, Kinberger, Garth A., Prakash, Thazha P., Seth, Punit P.
Formato: Artigo
Idioma:Inglês
Publicado em: Oxford University Press 2017
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Acesso em linha:https://ncbi.nlm.nih.gov/pmc/articles/PMC5716100/
https://ncbi.nlm.nih.gov/pubmed/29069408
https://ncbi.nlm.nih.govhttp://dx.doi.org/10.1093/nar/gkx960
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