[PDF][PDF] Characterizing the effect of GalNAc and phosphorothioate backbone on binding of antisense oligonucleotides to the asialoglycoprotein receptor
K Schmidt, TP Prakash, AJ Donner… - Nucleic acids …, 2017 - academic.oup.com
Targeted delivery of antisense oligonucleotides (ASO) to hepatocytes via the
asialoglycoprotein receptor (ASGR) has improved the potency of ASO drugs∼ 30-fold in the …
asialoglycoprotein receptor (ASGR) has improved the potency of ASO drugs∼ 30-fold in the …
Asialoglycoprotein receptor 1 mediates productive uptake of N-acetylgalactosamine-conjugated and unconjugated phosphorothioate antisense oligonucleotides into …
M Tanowitz, L Hettrick, A Revenko… - Nucleic acids …, 2017 - academic.oup.com
Antisense oligonucleotide (ASO) therapeutics show tremendous promise for the treatment of
previously intractable human diseases but to exert their effects on cellular RNA processing …
previously intractable human diseases but to exert their effects on cellular RNA processing …
Targeted delivery of antisense oligonucleotides to hepatocytes using triantennary N-acetyl galactosamine improves potency 10-fold in mice
TP Prakash, MJ Graham, J Yu, R Carty… - Nucleic acids …, 2014 - academic.oup.com
Triantennary N-acetyl galactosamine (GalNAc, GN3), a high-affinity ligand for the hepatocyte-
specific asialoglycoprotein receptor (ASGPR), enhances the potency of second-generation …
specific asialoglycoprotein receptor (ASGPR), enhances the potency of second-generation …
Conjugation of mono and di-GalNAc sugars enhances the potency of antisense oligonucleotides via ASGR mediated delivery to hepatocytes
GA Kinberger, TP Prakash, J Yu, G Vasquez… - Bioorganic & Medicinal …, 2016 - Elsevier
Antisense oligonucleotides (ASOs) conjugated to trivalent GalNAc ligands show 10-fold
enhanced potency for suppressing gene targets expressed in hepatocytes. Trivalent GalNAc …
enhanced potency for suppressing gene targets expressed in hepatocytes. Trivalent GalNAc …
Evaluation of the effect of 2′-O-methyl, fluoro hexitol, bicyclo and Morpholino nucleic acid modifications on potency of GalNAc conjugated antisense oligonucleotides …
TP Prakash, J Yu, GA Kinberger, A Low… - Bioorganic & Medicinal …, 2018 - Elsevier
The potency of antisense oligonucleotide (ASO) drugs has significantly improved in the
clinic after exploiting asialoglycoprotein receptor (ASGR) mediated delivery to hepatocytes …
clinic after exploiting asialoglycoprotein receptor (ASGR) mediated delivery to hepatocytes …
[HTML][HTML] Elucidation of the biotransformation pathways of a Galnac3-conjugated antisense oligonucleotide in rats and monkeys
CS Shemesh, ZY Rosie, HJ Gaus, S Greenlee… - … Therapy-Nucleic Acids, 2016 - cell.com
Triantennary N-acetyl galactosamine (GalNAc 3) is a high-affinity ligand for hepatocyte-
specific asialoglycoprotein receptors. Conjugation with GalNAc 3 via a trishexylamino (THA) …
specific asialoglycoprotein receptors. Conjugation with GalNAc 3 via a trishexylamino (THA) …
[HTML][HTML] Enhanced potency of GalNAc-conjugated antisense oligonucleotides in hepatocellular cancer models
Y Kim, M Jo, J Schmidt, X Luo, TP Prakash, T Zhou… - Molecular Therapy, 2019 - cell.com
Antisense oligonucleotides (ASOs) are a novel therapeutic approach to target difficult-to-
drug protein classes by targeting their corresponding mRNAs. Significantly enhanced ASO …
drug protein classes by targeting their corresponding mRNAs. Significantly enhanced ASO …
Integrated Assessment of the Clinical Performance of GalNAc3-Conjugated 2′-O-Methoxyethyl Chimeric Antisense Oligonucleotides: I. Human Volunteer …
ST Crooke, BF Baker, S Xia, RZ Yu, NJ Viney… - nucleic acid …, 2019 - liebertpub.com
Advances in medicinal chemistry have produced new chemical classes of antisense
oligonucleotides (ASOs) with enhanced therapeutic properties. Conjugation of the …
oligonucleotides (ASOs) with enhanced therapeutic properties. Conjugation of the …
Serial incorporation of a monovalent GalNAc phosphoramidite unit into hepatocyte-targeting antisense oligonucleotides
T Yamamoto, M Sawamura, F Wada… - Bioorganic & Medicinal …, 2016 - Elsevier
The targeting of abundant hepatic asialoglycoprotein receptors (ASGPR) with trivalent N-
acetylgalactosamine (GalNAc) is a reliable strategy for efficiently delivering antisense …
acetylgalactosamine (GalNAc) is a reliable strategy for efficiently delivering antisense …
Comprehensive Structure–Activity Relationship of Triantennary N-Acetylgalactosamine Conjugated Antisense Oligonucleotides for Targeted Delivery to Hepatocytes
TP Prakash, J Yu, MT Migawa… - Journal of medicinal …, 2016 - ACS Publications
The comprehensive structure–activity relationships of triantennary GalNAc conjugated ASOs
for enhancing potency via ASGR mediated delivery to hepatocytes is reported. Seventeen …
for enhancing potency via ASGR mediated delivery to hepatocytes is reported. Seventeen …
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