Impact of enhanced metabolic stability on pharmacokinetics and pharmacodynamics of GalNAc–siRNA conjugates

Clinical Proof of Concept for a Novel Hepatocyte-Targeting GalNAc-siRNA Conjugate

(2017) Tracy S. Zimmermann et al.   MOLECULAR THERAPY

A Highly Durable RNAi Therapeutic Inhibitor of PCSK9

(2017) Kevin Fitzgerald et al.   NEW ENGLAND JOURNAL OF MEDICINE

Well-Defined Multivalent Ligands for Hepatocytes Targeting via Asialoglycoprotein Receptor

(2016) Xiangang Huang et al.   BIOCONJUGATE CHEMISTRY

Synergistic effect of phosphorothioate, 5′-vinylphosphonate and GalNAc modifications for enhancing activity of synthetic siRNA

(2016) Thazha P. Prakash et al.   BIOORGANIC & MEDICINAL CHEMISTRY LETTERS

A convenient synthesis of 5′-triantennary N-acetyl-galactosamine clusters based on nitromethanetrispropionic acid

(2016) Michael T. Migawa et al.   BIOORGANIC & MEDICINAL CHEMISTRY LETTERS

5′-(E)-Vinylphosphonate: A Stable Phosphate Mimic Can Improve the RNAi Activity of siRNA-GalNAc Conjugates

(2016) Rubina Parmar et al.   CHEMBIOCHEM

Comprehensive Structure–Activity Relationship of Triantennary N-Acetylgalactosamine Conjugated Antisense Oligonucleotides for Targeted Delivery to Hepatocytes

(2016) Thazha P. Prakash et al.   JOURNAL OF MEDICINAL CHEMISTRY

An Investigational RNAi Therapeutic Targeting Glycolate Oxidase Reduces Oxalate Production in Models of Primary Hyperoxaluria

(2016) Abigail Liebow et al.   JOURNAL OF THE AMERICAN SOCIETY OF NEPHROLOGY

siRNA carrying an (E)-vinylphosphonate moiety at the 5΄ end of the guide strand augments gene silencing by enhanced binding to human Argonaute-2

(2016) Elad Elkayam et al.   NUCLEIC ACIDS RESEARCH

Disposition and Pharmacokinetics of a GalNAc3-Conjugated Antisense Oligonucleotide Targeting Human Lipoprotein (a) in Monkeys

(2016) Rosie Z. Yu et al.   Nucleic Acid Therapeutics

Disposition and Pharmacology of a GalNAc3-conjugated ASO Targeting Human Lipoprotein (a) in Mice

(2016) Rosie Z Yu et al.   Molecular Therapy-Nucleic Acids

siRNA Conjugates Carrying Sequentially Assembled Trivalent N-Acetylgalactosamine Linked Through Nucleosides Elicit Robust Gene Silencing In Vivo in Hepatocytes

(2015) Shigeo Matsuda et al.   ACS Chemical Biology

Hepatocyte-Specific Delivery of siRNAs Conjugated to Novel Non-nucleosidic TrivalentN-Acetylgalactosamine Elicits Robust Gene Silencing in Vivo

(2015) Kallanthottathil G. Rajeev et al.   CHEMBIOCHEM

An RNAi therapeutic targeting antithrombin to rebalance the coagulation system and promote hemostasis in hemophilia

(2015) Alfica Sehgal et al.   NATURE MEDICINE

Knocking down disease: a progress report on siRNA therapeutics

(2015) Anders Wittrup et al.   NATURE REVIEWS GENETICS

Preclinical Development of a Subcutaneous ALAS1 RNAi Therapeutic for Treatment of Hepatic Porphyrias Using Circulating RNA Quantification

(2015) Amy Chan et al.   Molecular Therapy-Nucleic Acids

Multivalent N-Acetylgalactosamine-Conjugated siRNA Localizes in Hepatocytes and Elicits Robust RNAi-Mediated Gene Silencing

(2014) Jayaprakash K. Nair et al.   JOURNAL OF THE AMERICAN CHEMICAL SOCIETY

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

Quantitative evaluation of siRNA delivery in vivo

(2010) Y. Pei et al.   RNA

Reactivity of 3H-1,2,4-dithiazole-3-thiones and 3H-1,2-dithiole-3-thiones as sulfurizing agents for oligonucleotide synthesis

(2010) Andrei P. Guzaev   TETRAHEDRON LETTERS

Synthesis and characterization of 2′-modified-4′-thioRNA: a comprehensive comparison of nuclease stability

(2009) Mayumi Takahashi et al.   NUCLEIC ACIDS RESEARCH