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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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Detalles Bibliográficos
Autores principales: Tanowitz, Michael, Hettrick, Lisa, Revenko, Alexey, Kinberger, Garth A., Prakash, Thazha P., Seth, Punit P.
Formato: Online Artículo Texto
Lenguaje:English
Publicado: Oxford University Press 2017
Materias:
Acceso en línea:https://www.ncbi.nlm.nih.gov/pmc/articles/PMC5716100/
https://www.ncbi.nlm.nih.gov/pubmed/29069408
http://dx.doi.org/10.1093/nar/gkx960