DNA/RNA heteroduplex oligonucleotide for highly efficient gene silencing

Preclinical and clinical development of siRNA-based therapeutics

(2015) Gulnihal Ozcan et al.   ADVANCED DRUG DELIVERY REVIEWS

Clinical and Preclinical Pharmacokinetics and Pharmacodynamics of Mipomersen (Kynamro®): A Second-Generation Antisense Oligonucleotide Inhibitor of Apolipoprotein B

(2015) Richard S. Geary et al.   CLINICAL PHARMACOKINETICS

Chimeric Antisense Oligonucleotide Conjugated to α-Tocopherol

(2015) Tomoko Nishina et al.   Molecular Therapy-Nucleic Acids

Preclinical Evaluation of the Toxicological Effects of a Novel Constrained Ethyl Modified Antisense Compound Targeting Signal Transducer and Activator of Transcription 3 in Mice and Cynomolgus Monkeys

(2015) Sebastien A. Burel et al.   Nucleic Acid Therapeutics

Current issues of RNAi therapeutics delivery and development

(2014) D. Haussecker   JOURNAL OF CONTROLLED RELEASE

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

Non-viral vectors for gene-based therapy

(2014) Hao Yin et al.   NATURE REVIEWS GENETICS

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

Degradable lipid nanoparticles with predictable in vivo siRNA delivery activity

(2014) Kathryn A. Whitehead et al.   Nature Communications

Shielding of Lipid Nanoparticles for siRNA Delivery: Impact on Physicochemical Properties, Cytokine Induction, and Efficacy

(2014) Varun Kumar et al.   Molecular Therapy-Nucleic Acids

A Kinetic Model Explains Why Shorter and Less Affine Enzyme-recruiting Oligonucleotides Can Be More Potent

(2014) Lykke Pedersen et al.   Molecular Therapy-Nucleic Acids

Lipid Nanoparticles Improve Activity of Single-Stranded siRNA and Gapmer Antisense Oligonucleotides in Animals

(2013) Thazha P. Prakash et al.   ACS Chemical Biology

Effect of an RNA interference drug on the synthesis of proprotein convertase subtilisin/kexin type 9 (PCSK9) and the concentration of serum LDL cholesterol in healthy volunteers: a randomised, single-blind, placebo-controlled, phase 1 trial

(2013) Kevin Fitzgerald et al.   LANCET

An antisense oligonucleotide against SOD1 delivered intrathecally for patients with SOD1 familial amyotrophic lateral sclerosis: a phase 1, randomised, first-in-man study

(2013) Timothy M Miller et al.   LANCET NEUROLOGY

Image-based analysis of lipid nanoparticle–mediated siRNA delivery, intracellular trafficking and endosomal escape

(2013) Jerome Gilleron et al.   NATURE BIOTECHNOLOGY

Delivery materials for siRNA therapeutics

(2013) Rosemary Kanasty et al.   NATURE MATERIALS

Biotech comes to its 'antisenses' after hard-won drug approval

(2013) Kevin Jiang   NATURE MEDICINE

Safety and Efficacy of RNAi Therapy for Transthyretin Amyloidosis

(2013) Teresa Coelho et al.   NEW ENGLAND JOURNAL OF MEDICINE

Comparison of Hepatic Transcription Profiles of Locked Ribonucleic Acid Antisense Oligonucleotides: Evidence of Distinct Pathways Contributing to Non-target Mediated Toxicity in Mice

(2013) Satoko Kakiuchi-Kiyota et al.   TOXICOLOGICAL SCIENCES

Lipid Conjugated Oligonucleotides: A Useful Strategy for Delivery

(2012) Mouna Raouane et al.   BIOCONJUGATE CHEMISTRY

Clinical pharmacological properties of mipomersen (Kynamro), a second generation antisense inhibitor of apolipoprotein B

(2012) Stanley T. Crooke et al.   BRITISH JOURNAL OF CLINICAL PHARMACOLOGY

TricycloDNA-modified oligo-2′-deoxyribonucleotides reduce scavenger receptor B1 mRNA in hepatic and extra-hepatic tissues—a comparative study of oligonucleotide length, design and chemistry

(2012) Sue Murray et al.   NUCLEIC ACIDS RESEARCH

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

Selection, Optimization, and Pharmacokinetic Properties of a Novel, Potent Antiviral Locked Nucleic Acid-Based Antisense Oligomer Targeting Hepatitis C Virus Internal Ribosome Entry Site

(2011) Carl Laxton et al.   ANTIMICROBIAL AGENTS AND CHEMOTHERAPY

Cellular Uptake and Intracellular Trafficking of Antisense and siRNA Oligonucleotides

(2011) Rudolph L. Juliano et al.   BIOCONJUGATE CHEMISTRY

Hybrid lipid oligonucleotide conjugates: synthesis, self-assemblies and biomedical applications

(2011) Amit Patwa et al.   CHEMICAL SOCIETY REVIEWS

Efficient In Vivo Delivery of siRNA Into Brain Capillary Endothelial Cells Along With Endogenous Lipoprotein

(2011) Hiroya Kuwahara et al.   MOLECULAR THERAPY

Mechanisms of single-stranded phosphorothioate modified antisense oligonucleotide accumulation in hepatocytes

(2011) Erich Koller et al.   NUCLEIC ACIDS RESEARCH

rnaset2 mutant zebrafish model familial cystic leukoencephalopathy and reveal a role for RNase T2 in degrading ribosomal RNA

(2011) N. Haud et al.   PROCEEDINGS OF THE NATIONAL ACADEMY OF SCIENCES OF THE UNITED STATES OF AMERICA

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

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

High-Density Lipoprotein Facilitates In Vivo Delivery of α-Tocopherol–Conjugated Short-Interfering RNA to the Brain

(2010) Yoshitaka Uno et al.   HUMAN GENE THERAPY

Lipophilic siRNAs mediate efficient gene silencing in oligodendrocytes with direct CNS delivery

(2010) Qingmin Chen et al.   JOURNAL OF CONTROLLED RELEASE

Synthesis and Biophysical Evaluation of 2′,4′-Constrained 2′O-Methoxyethyl and 2′,4′-Constrained 2′O-Ethyl Nucleic Acid Analogues

(2010) Punit P. Seth et al.   JOURNAL OF ORGANIC CHEMISTRY

Targeted Delivery of RNAi Therapeutics With Endogenous and Exogenous Ligand-Based Mechanisms

(2010) Akin Akinc et al.   MOLECULAR THERAPY

Rational design of cationic lipids for siRNA delivery

(2010) Sean C Semple et al.   NATURE BIOTECHNOLOGY

Short locked nucleic acid antisense oligonucleotides potently reduce apolipoprotein B mRNA and serum cholesterol in mice and non-human primates

(2010) Ellen Marie Straarup et al.   NUCLEIC ACIDS RESEARCH

The carboxy-terminal fragment of α1A calcium channel preferentially aggregates in the cytoplasm of human spinocerebellar ataxia type 6 Purkinje cells

(2009) Taro Ishiguro et al.   ACTA NEUROPATHOLOGICA

Efficient gene silencing by delivery of locked nucleic acid antisense oligonucleotides, unassisted by transfection reagents

(2009) C. A. Stein et al.   NUCLEIC ACIDS RESEARCH

Cross-species comparison of in vivo PK/PD relationships for second-generation antisense oligonucleotides targeting apolipoprotein B-100

(2008) Rosie Z. Yu et al.   BIOCHEMICAL PHARMACOLOGY

Efficient In Vivo Delivery of siRNA to the Liver by Conjugation of α-Tocopherol

(2008) Kazutaka Nishina et al.   MOLECULAR THERAPY