Conjugation of mono and di-GalNAc sugars enhances the potency of antisense oligonucleotides via ASGR mediated delivery to hepatocytes

Bioorganic and Medicinal Chemistry Letters

Volumn 26, Issue 15, 2016, Pages 3690-3693

  Kinberger, Garth A ^a   Prakash, Thazha P ^a   Yu, Jinghua ^a   Vasquez, Guillermo ^a   Low, Audrey ^a   Chappell, Alfred ^a   Schmidt, Karsten ^a   Murray, Heather M ^a   Gaus, Hans ^a   Swayze, Eric E ^a   Seth, Punit P ^a  

^a ISIS PHARMACEUTICALS   (United States)

Author keywords

ASGR; ASO; Delivery; GalNAc; Hepatocytes

Indexed keywords

ANTISENSE OLIGONUCLEOTIDE; ASIALOGLYCOPROTEIN RECEPTOR; MESSENGER RNA; N ACETYLGALACTOSAMINE; SCARB1 PROTEIN, MOUSE; SCAVENGER RECEPTOR B;

ARTICLE; BINDING AFFINITY; CHEMICAL MODIFICATION; DRUG CONJUGATION; DRUG POTENCY; DRUG RECEPTOR BINDING; LIVER CELL; MOUSE; NONHUMAN; ANIMAL; ANTAGONISTS AND INHIBITORS; C57BL MOUSE; CHEMISTRY; CONFORMATION; DOSE RESPONSE; DRUG EFFECTS; METABOLISM; STRUCTURE ACTIVITY RELATION;

ACETYLGALACTOSAMINE; ANIMALS; ASIALOGLYCOPROTEIN RECEPTOR; DOSE-RESPONSE RELATIONSHIP, DRUG; HEPATOCYTES; MICE; MICE, INBRED C57BL; MOLECULAR CONFORMATION; OLIGONUCLEOTIDES, ANTISENSE; RNA, MESSENGER; SCAVENGER RECEPTORS, CLASS B; STRUCTURE-ACTIVITY RELATIONSHIP;

EID: 84971665279     PISSN: 0960894X     EISSN: 14643405     Source Type: Journal    
DOI: 10.1016/j.bmcl.2016.05.084     Document Type: Article

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