New Oligonucleotide 2′-O-Alkyl N3′→P5′ (Thio)-Phosphoramidates as Potent Antisense Agents: Physicochemical Properties and Biological Activity

Antisense Technology: A Review

(2021) Stanley T. Crooke et al.   JOURNAL OF BIOLOGICAL CHEMISTRY

Stereochemistry of internucleoside phosphorus atom affects sugar pucker and acid hydrolysis of N3′-P5′ thio-phosphoramidates

(2021) Soheil Pourshahian et al.   TETRAHEDRON LETTERS

Randomized, Single-Blind, Multicenter Phase II Study of Two Doses of Imetelstat in Relapsed or Refractory Myelofibrosis

(2021) John Mascarenhas et al.   JOURNAL OF CLINICAL ONCOLOGY

The current landscape of nucleic acid therapeutics

(2021) Jayesh A. Kulkarni et al.   Nature Nanotechnology

Advances in oligonucleotide drug delivery

(2020) Thomas C. Roberts et al.   NATURE REVIEWS DRUG DISCOVERY

Imetelstat Achieves Meaningful and Durable Transfusion Independence in High Transfusion–Burden Patients With Lower-Risk Myelodysplastic Syndromes in a Phase II Study

(2020) David P. Steensma et al.   JOURNAL OF CLINICAL ONCOLOGY

Nonclinical Exon Skipping Studies with 2′-O-Methyl Phosphorothioate Antisense Oligonucleotides in mdx and mdx-utrn−/− Mice Inspired by Clinical Trial Results

(2019) Maaike van Putten et al.   Nucleic Acid Therapeutics

Fatty acid conjugation enhances potency of antisense oligonucleotides in muscle

(2019) Thazha P Prakash et al.   NUCLEIC ACIDS RESEARCH

Sequencing of Phosphoramidate Oligonucleotides by Acid Hydrolysis and Mass Spectrometry

(2019) Soheil Pourshahian et al.   ANALYTICAL CHEMISTRY

Adjusting the Structure of 2′-Modified Nucleosides and Oligonucleotides via C4′-α-F or C4′-α-OMe Substitution: Synthesis and Conformational Analysis

(2018) Elise Malek-Adamian et al.   JOURNAL OF ORGANIC CHEMISTRY

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

Application of 2′-O-(2-N-Methylcarbamoylethyl) Nucleotides in RNase H-Dependent Antisense Oligonucleotides

(2018) Yoshiaki Masaki et al.   Nucleic Acid Therapeutics

Cellular uptake and trafficking of antisense oligonucleotides

(2017) Stanley T Crooke et al.   NATURE BIOTECHNOLOGY

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

(2017) Xiulong Shen et al.   NUCLEIC ACIDS RESEARCH

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

Carrier-free Gene Silencing by Amphiphilic Nucleic Acid Conjugates in Differentiated Intestinal Cells

(2016) Elena Moroz et al.   Molecular Therapy-Nucleic Acids

Pharmacokinetics, biodistribution and cell uptake of antisense oligonucleotides

(2015) Richard S. Geary et al.   ADVANCED DRUG DELIVERY REVIEWS

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

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

Surprising Base Pairing and Structural Properties of 2′-Trifluoromethylthio-Modified Ribonucleic Acids

(2014) Marija Košutić et al.   JOURNAL OF THE AMERICAN CHEMICAL SOCIETY

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