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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Vydáno v:Nucleic Acids Res
Hlavní autoři: Tanowitz, Michael, Hettrick, Lisa, Revenko, Alexey, Kinberger, Garth A., Prakash, Thazha P., Seth, Punit P.
Médium: Artigo
Jazyk:Inglês
Vydáno: Oxford University Press 2017
Témata:
Molecular Biology
On-line přístup: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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