N-Acetyl Galactosamine Targeting: Paving the Way for Clinical Application of Nucleotide Medicines in Cardiovascular Diseases

The growth of siRNA-based therapeutics: updated clinical studies

(2021) M. May Zhang et al.   BIOCHEMICAL PHARMACOLOGY

Hepatic Lactate Dehydrogenase A is an RNA Interference Target for the Treatment of All Known Types of Primary Hyperoxaluria

(2021) Gema Ariceta et al.   Kidney International Reports

Nonclinical Pharmacokinetics and ADME of Givosiran, the First Approved GalNAc-conjugated RNAi Therapeutic

(2021) Jing Li et al.   DRUG METABOLISM AND DISPOSITION

Phase 3 Multicenter Study of Revusiran in Patients with Hereditary Transthyretin-Mediated (hATTR) Amyloidosis with Cardiomyopathy (ENDEAVOUR)

(2020) Daniel P. Judge et al.   CARDIOVASCULAR DRUGS AND THERAPY

Eosinophil and mast cell Siglecs: From biology to drug target

(2020) Jeremy A. O'Sullivan et al.   JOURNAL OF LEUKOCYTE BIOLOGY

Lipoprotein(a) Reduction in Persons with Cardiovascular Disease

(2020) Sotirios Tsimikas et al.   NEW ENGLAND JOURNAL OF MEDICINE

A Meta-Analysis of Cardiovascular Outcomes in Patients With Hypercholesterolemia Treated With Inclisiran

(2020) Abdul Aziz A. Asbeutah et al.   AMERICAN JOURNAL OF CARDIOLOGY

Gene-based therapy in lipid management: the winding road from promise to practice

(2020) Tycho R. Tromp et al.   EXPERT OPINION ON INVESTIGATIONAL DRUGS

Two Phase 3 Trials of Inclisiran in Patients with Elevated LDL Cholesterol

(2020) Kausik K. Ray et al.   NEW ENGLAND JOURNAL OF MEDICINE

Recent advances in siRNA delivery mediated by lipid-based nanoparticles

(2020) Sei Yonezawa et al.   ADVANCED DRUG DELIVERY REVIEWS

Delivery of Oligonucleotides to the Liver with GalNAc: From Research to Registered Therapeutic Drug

(2020) Alexandre J. Debacker et al.   MOLECULAR THERAPY

Phase 3 Trial of RNAi Therapeutic Givosiran for Acute Intermittent Porphyria

(2020) Manisha Balwani et al.   NEW ENGLAND JOURNAL OF MEDICINE

Vupanorsen, an N-acetyl galactosamine-conjugated antisense drug to ANGPTL3 mRNA, lowers triglycerides and atherogenic lipoproteins in patients with diabetes, hepatic steatosis, and hypertriglyceridaemia

(2020) Daniel Gaudet et al.   EUROPEAN HEART JOURNAL

Investigating the pharmacodynamic durability of GalNAc–siRNA conjugates

(2020) Christopher R Brown et al.   NUCLEIC ACIDS RESEARCH

Selective tumor antigen vaccine delivery to human CD169+ antigen-presenting cells using ganglioside-liposomes

(2020) Alsya J. Affandi et al.   PROCEEDINGS OF THE NATIONAL ACADEMY OF SCIENCES OF THE UNITED STATES OF AMERICA

In Vivo siRNA Delivery to Immunosuppressive Liver Macrophages by α-Mannosyl-Functionalized Cationic Nanohydrogel Particles

(2020) Leonard Kaps et al.   Cells

N-acetyl galactosamine-conjugated antisense drug to APOC3 mRNA, triglycerides and atherogenic lipoprotein levels

(2019) Veronica J Alexander et al.   EUROPEAN HEART JOURNAL

A Sulfonamide Sialoside Analogue for Targeting Siglec-8 and -F on Immune Cells

(2019) Corwin M. Nycholat et al.   JOURNAL OF THE AMERICAN CHEMICAL SOCIETY

Current Position on the Role of Monomeric C-reactive Protein in Vascular Pathology and Atherothrombosis

(2019) Ivan S. Melnikov et al.   CURRENT PHARMACEUTICAL DESIGN

The Onpattro story and the clinical translation of nanomedicines containing nucleic acid-based drugs

(2019) Akin Akinc et al.   Nature Nanotechnology

Evaluation of GalNAc-siRNA Conjugate Activity in Pre-clinical Animal Models with Reduced Asialoglycoprotein Receptor Expression

(2018) Jennifer L.S. Willoughby et al.   MOLECULAR THERAPY

GalNAc-siRNA Conjugates: Leading the Way for Delivery of RNAi Therapeutics

(2018) Aaron D. Springer et al.   Nucleic Acid Therapeutics

Therapeutic Oligonucleotides: State of the Art

(2018) C.I. Edvard Smith et al.   Annual Review of Pharmacology and Toxicology

MicroRNAs as Regulators of Insulin Signaling: Research Updates and Potential Therapeutic Perspectives in Type 2 Diabetes

(2018) Laura Nigi et al.   INTERNATIONAL JOURNAL OF MOLECULAR SCIENCES

Highly specific delivery of siRNA to hepatocytes circumvents endothelial cell-mediated lipid nanoparticle-associated toxicity leading to the safe and efficacious decrease in the hepatitis B virus

(2017) Yusuke Sato et al.   JOURNAL OF CONTROLLED RELEASE

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

Targeting of Antithrombin in Hemophilia A or B with RNAi Therapy

(2017) K. John Pasi et al.   NEW ENGLAND JOURNAL OF MEDICINE

Cardiovascular and Metabolic Effects of ANGPTL3 Antisense Oligonucleotides

(2017) Mark J. Graham et al.   NEW ENGLAND JOURNAL OF MEDICINE

The delivery of therapeutic oligonucleotides

(2016) Rudolph L. Juliano   NUCLEIC ACIDS RESEARCH

Inhibition of insulin-like growth factor II (IGF-II)-dependent cell growth by multidentate pentamannosyl 6-phosphate-based ligands targeting the mannose 6-phosphate/IGF-II receptor

(2016) Megan E. Zavorka et al.   Oncotarget

Cell Type-Specific Delivery of RNAi by Ligand-Functionalized Curdlan Nanoparticles: Balancing the Receptor Mediation and the Charge Motivation

(2015) Yinga Wu et al.   ACS Applied Materials & Interfaces

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

Reductively Responsive Hydrogel Nanoparticles with Uniform Size, Shape, and Tunable Composition for Systemic siRNA Delivery in Vivo

(2015) Da Ma et al.   MOLECULAR PHARMACEUTICS

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

Scavenger Receptor-AI–Targeted Iron Oxide Nanoparticles for In Vivo MRI Detection of Atherosclerotic Lesions

(2013) Filip M.E. Segers et al.   ARTERIOSCLEROSIS THROMBOSIS AND VASCULAR BIOLOGY

Identification of a novel CD40 ligand for targeted imaging of inflammatory plaques by phage display

(2013) Haixiang Yu et al.   FASEB JOURNAL

Liver as a target for oligonucleotide therapeutics

(2013) Alfica Sehgal et al.   JOURNAL OF HEPATOLOGY

Macrophage-Specific RNA Interference Targeting via “Click”, Mannosylated Polymeric Micelles

(2013) Shann S. Yu et al.   MOLECULAR PHARMACEUTICS

Design and Validation of a Specific Scavenger Receptor Class AI Binding Peptide for Targeting the Inflammatory Atherosclerotic Plaque

(2012) Filip M.E. Segers et al.   ARTERIOSCLEROSIS THROMBOSIS AND VASCULAR BIOLOGY

NMR study of ligand release from asialoglycoprotein receptor under solution conditions in early endosomes

(2012) Takuo Onizuka et al.   FEBS Journal

RNAi-based therapeutic strategies for metabolic disease

(2011) Michael P. Czech et al.   Nature Reviews Endocrinology

RNA Targeting Therapeutics: Molecular Mechanisms of Antisense Oligonucleotides as a Therapeutic Platform

(2010) C. Frank Bennett et al.   Annual Review of Pharmacology and Toxicology