Title : Characterizing the effect of GalNAc and phosphorothioate backbone on binding of antisense oligonucleotides to the asialoglycoprotein receptor.

Pub. Date : 2017 Mar 17

PMID : 28158620






4 Functional Relationships(s)
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1 Characterizing the effect of GalNAc and phosphorothioate backbone on binding of antisense oligonucleotides to the asialoglycoprotein receptor. N-acetylgalactosaminuronic acid asialoglycoprotein receptor 1 Mus musculus
2 Two and three GalNAc conjugated single stranded ASOs bind the ASGR with the strongest affinity and display optimal in vitro and in vivo activities. N-acetylgalactosaminuronic acid asialoglycoprotein receptor 1 Mus musculus
3 1 GalNAc conjugated ASOs showed 10-fold reduced ASGR binding affinity relative to three GalNAc ASOs but only 2-fold reduced activity in mice. N-acetylgalactosaminuronic acid asialoglycoprotein receptor 1 Mus musculus
4 An unexpected observation was that the ASGR also appears to play a role in the uptake of unconjugated phosphorothioate modified ASOs in the liver as evidenced by the loss of activity of GalNAc conjugated and unconjugated ASOs in ASGR knockout mice. N-acetylgalactosaminuronic acid asialoglycoprotein receptor 1 Mus musculus