Effect of modular conjugation strategy for N-acetylgalactosamine-targeted antisense oligonucleotides

Cholesterol–GalNAc Dual Conjugation Strategy for Reducing Renal Distribution of Antisense Oligonucleotides

(2018) Fumito Wada et al.   Nucleic Acid Therapeutics

Receptor-Mediated Uptake of Phosphorothioate Antisense Oligonucleotides in Different Cell Types of the Liver

(2018) Colton M. Miller et al.   Nucleic Acid Therapeutics

A Sensitive In Vitro Approach to Assess the Hybridization-Dependent Toxic Potential of High Affinity Gapmer Oligonucleotides

(2018) Andreas Dieckmann et al.   Molecular Therapy-Nucleic Acids

Chemical Modification Study of Antisense Gapmers

(2018) Robert Stanton et al.   Nucleic Acid Therapeutics

RNA Reduction and Hepatotoxic Potential Caused by Non-Gapmer Antisense Oligonucleotides

(2018) Shin-Ichiro Hori et al.   Nucleic Acid Therapeutics

The chemical evolution of oligonucleotide therapies of clinical utility

(2017) Anastasia Khvorova et al.   NATURE BIOTECHNOLOGY

Overcoming cellular barriers for RNA therapeutics

(2017) Steven F Dowdy   NATURE BIOTECHNOLOGY

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

(2017) Karsten Schmidt et al.   NUCLEIC ACIDS RESEARCH

Chemistry, mechanism and clinical status of antisense oligonucleotides and duplex RNAs

(2017) Xiulong Shen et al.   NUCLEIC ACIDS RESEARCH

Preclinical and Clinical Advances of GalNAc-Decorated Nucleic Acid Therapeutics

(2017) Yuanyu Huang   Molecular Therapy-Nucleic Acids

Serial incorporation of a monovalent GalNAc phosphoramidite unit into hepatocyte-targeting antisense oligonucleotides

(2016) Tsuyoshi Yamamoto et al.   BIOORGANIC & MEDICINAL CHEMISTRY

Development of Antisense Drugs for Dyslipidemia

(2016) Tsuyoshi Yamamoto et al.   Journal of Atherosclerosis and Thrombosis

The Medicinal Chemistry of Therapeutic Oligonucleotides

(2016) W. Brad Wan et al.   JOURNAL OF MEDICINAL CHEMISTRY

Elucidation of the Biotransformation Pathways of a Galnac3-conjugated Antisense Oligonucleotide in Rats and Monkeys

(2016) Colby S Shemesh et al.   Molecular Therapy-Nucleic Acids

Ribonuclease H1-dependent hepatotoxicity caused by locked nucleic acid-modified gapmer antisense oligonucleotides

(2016) Takeshi Kasuya et al.   Scientific Reports

Efficient Synthesis and Biological Evaluation of 5′-GalNAc Conjugated Antisense Oligonucleotides

(2015) Michael E. Østergaard et al.   BIOCONJUGATE CHEMISTRY

Antisense-mediated reduction of proprotein convertase subtilisin/kexin type 9 (PCSK9): a first-in-human randomized, placebo-controlled trial

(2015) Eveline P. van Poelgeest et al.   BRITISH JOURNAL OF CLINICAL PHARMACOLOGY

Sequence motifs associated with hepatotoxicity of locked nucleic acid—modified antisense oligonucleotides

(2014) Andrew D. Burdick et al.   NUCLEIC ACIDS RESEARCH

Targeted delivery of antisense oligonucleotides to hepatocytes using triantennaryN-acetyl galactosamine improves potency 10-fold in mice

(2014) Thazha P. Prakash et al.   NUCLEIC ACIDS RESEARCH

Hepatotoxic Potential of Therapeutic Oligonucleotides Can Be Predicted from Their Sequence and Modification Pattern

(2013) Peter H. Hagedorn et al.   Nucleic Acid Therapeutics

Structure Activity Relationships of α-L-LNA Modified Phosphorothioate Gapmer Antisense Oligonucleotides in Animals

(2012) Punit P Seth et al.   Molecular Therapy-Nucleic Acids

Cholesterol-lowering Action of BNA-based Antisense Oligonucleotides Targeting PCSK9 in Atherogenic Diet-induced Hypercholesterolemic Mice

(2012) Tsuyoshi Yamamoto et al.   Molecular Therapy-Nucleic Acids

Antisense drug discovery and development

(2011) Tsuyoshi Yamamoto et al.   Future Medicinal Chemistry

Trivalent, Gal/GalNAc-containing ligands designed for the asialoglycoprotein receptor

(2008) Oleg Khorev et al.   BIOORGANIC & MEDICINAL CHEMISTRY

The Asialoglycoprotein Receptor Regulates Levels of Plasma Glycoproteins Terminating with Sialic Acid α2,6-Galactose

(2008) Lindsay M. Steirer et al.   JOURNAL OF BIOLOGICAL CHEMISTRY