Efficient Gene Suppression by DNA/DNA Double-Stranded Oligonucleotide In Vivo

Quantitative Mapping of Endosomal DNA Processing by Single Molecule Counting

(2019) Ved Prakash et al.   ANGEWANDTE CHEMIE-INTERNATIONAL EDITION

Highly efficient silencing of microRNA by heteroduplex oligonucleotides

(2019) Kotaro Yoshioka et al.   NUCLEIC ACIDS RESEARCH

Advances in the treatment of hereditary transthyretin amyloidosis: A review

(2019) Morie A. Gertz et al.   Brain and Behavior

RNA-Targeted Therapeutics

(2018) Stanley T. Crooke et al.   Cell Metabolism

Nusinersen versus Sham Control in Later-Onset Spinal Muscular Atrophy

(2018) Eugenio Mercuri et al.   NEW ENGLAND JOURNAL OF MEDICINE

Acute hepatotoxicity of 2′ fluoro-modified 5–10–5 gapmer phosphorothioate oligonucleotides in mice correlates with intracellular protein binding and the loss of DBHS proteins

(2018) Wen Shen et al.   NUCLEIC ACIDS RESEARCH

Cellular uptake mediated by epidermal growth factor receptor facilitates the intracellular activity of phosphorothioate-modified antisense oligonucleotides

(2018) Shiyu Wang et al.   NUCLEIC ACIDS RESEARCH

Modulation of blood-brain barrier function by a heteroduplex oligonucleotide in vivo

(2018) Hiroya Kuwahara et al.   Scientific Reports

Artificial cationic oligosaccharides for heteroduplex oligonucleotide-type drugs

(2018) Rintaro Iwata Hara et al.   Scientific Reports

Cellular uptake and trafficking of antisense oligonucleotides

(2017) Stanley T Crooke et al.   NATURE BIOTECHNOLOGY

Nusinersen versus Sham Control in Infantile-Onset Spinal Muscular Atrophy

(2017) Richard S. Finkel et al.   NEW ENGLAND JOURNAL OF MEDICINE

Asialoglycoprotein receptor 1 mediates productive uptake of N-acetylgalactosamine-conjugated and unconjugated phosphorothioate antisense oligonucleotides into liver hepatocytes

(2017) Michael Tanowitz et al.   NUCLEIC ACIDS RESEARCH

Stabilin-1 and Stabilin-2 are specific receptors for the cellular internalization of phosphorothioate-modified antisense oligonucleotides (ASOs) in the liver

(2016) Colton M. Miller et al.   NUCLEIC ACIDS RESEARCH

Pharmacokinetic and Pharmacodynamic Investigations of ION-353382, a Model Antisense Oligonucleotide: Using Alpha-2-Macroglobulin and Murinoglobulin Double-Knockout Mice

(2016) Colby S. Shemesh et al.   Nucleic Acid Therapeutics

Toll-like receptor 8 senses degradation products of single-stranded RNA

(2015) Hiromi Tanji et al.   NATURE STRUCTURAL & MOLECULAR BIOLOGY

DNA/RNA heteroduplex oligonucleotide for highly efficient gene silencing

(2015) Kazutaka Nishina et al.   Nature Communications

Toll-like receptor 8 senses degradation products of single-stranded RNA

(2015) Hiromi Tanji et al.   NATURE STRUCTURAL & MOLECULAR BIOLOGY

Chimeric Antisense Oligonucleotide Conjugated to α-Tocopherol

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

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

Bacterial RNA:DNA hybrids are activators of the NLRP3 inflammasome

(2014) S. Kailasan Vanaja et al.   PROCEEDINGS OF THE NATIONAL ACADEMY OF SCIENCES OF THE UNITED STATES OF AMERICA

Eteplirsen for the treatment of Duchenne muscular dystrophy

(2013) Jerry R. Mendell et al.   ANNALS OF NEUROLOGY

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

A rapid two-step method for isolation of functional primary mouse hepatocytes: cell characterization and asialoglycoprotein receptor based assay development

(2011) Mariano Severgnini et al.   CYTOTECHNOLOGY

Antisense correction of SMN2 splicing in the CNS rescues necrosis in a type III SMA mouse model

(2010) Y. Hua et al.   GENES & DEVELOPMENT

Ribonuclease H: the enzymes in eukaryotes

(2009) Susana M. Cerritelli et al.   FEBS Journal

Short Antisense Oligonucleotides with Novel 2′−4′ Conformationaly Restricted Nucleoside Analogues Show Improved Potency without Increased Toxicity in Animals

(2008) Punit P. Seth et al.   JOURNAL OF MEDICINAL CHEMISTRY