Synthesis, chirality-dependent conformational and biological properties of siRNAs containing 5′-(R)- and 5′-(S)-C-methyl-guanosine

Inclisiran for the Treatment of Heterozygous Familial Hypercholesterolemia

(2020) Frederick J. Raal et al.   NEW ENGLAND JOURNAL OF MEDICINE

Phase 3 Trial of RNAi Therapeutic Givosiran for Acute Intermittent Porphyria

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

Treating Disease at the RNA Level with Oligonucleotides

(2019) Arthur A. Levin   NEW ENGLAND JOURNAL OF MEDICINE

Safety evaluation of 2′-deoxy-2′-fluoro nucleotides in GalNAc-siRNA conjugates

(2019) Maja M Janas et al.   NUCLEIC ACIDS RESEARCH

Phase 1 Trial of an RNA Interference Therapy for Acute Intermittent Porphyria

(2019) Eliane Sardh et al.   NEW ENGLAND JOURNAL OF MEDICINE

Re-Engineering RNA Molecules into Therapeutic Agents

(2019) Martin Egli et al.   ACCOUNTS OF CHEMICAL RESEARCH

The cGAS-cGAMP-STING Pathway: A Molecular Link Between Immunity and Metabolism

(2019) Juli Bai et al.   DIABETES

siRNA therapeutics: a clinical reality

(2019) Phei Er Saw et al.   Science China-Life Sciences

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

(2019) Akin Akinc et al.   Nature Nanotechnology

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

Advanced siRNA Designs Further Improve In Vivo Performance of GalNAc-siRNA Conjugates

(2018) Donald J. Foster et al.   MOLECULAR THERAPY

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

Structural basis for the synergy of 4′- and 2′-modifications on siRNA nuclease resistance, thermal stability and RNAi activity

(2018) Joel M Harp et al.   NUCLEIC ACIDS RESEARCH

4′-C-Methoxy-2′-deoxy-2′-fluoro Modified Ribonucleotides Improve Metabolic Stability and Elicit Efficient RNAi-Mediated Gene Silencing

(2017) Elise Malek-Adamian et al.   JOURNAL OF THE AMERICAN CHEMICAL SOCIETY

Oligonucleotide Therapeutics — A New Class of Cholesterol-Lowering Drugs

(2017) Anastasia Khvorova   NEW ENGLAND JOURNAL OF MEDICINE

A Highly Durable RNAi Therapeutic Inhibitor of PCSK9

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

Inclisiran in Patients at High Cardiovascular Risk with Elevated LDL Cholesterol

(2017) Kausik K. Ray et al.   NEW ENGLAND JOURNAL OF MEDICINE

Structural Basis of Duplex Thermodynamic Stability and Enhanced Nuclease Resistance of 5′-C-Methyl Pyrimidine-Modified Oligonucleotides

(2016) Alexander V. Kel′in et al.   JOURNAL OF ORGANIC CHEMISTRY

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

(2015) Alfica Sehgal et al.   NATURE MEDICINE

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

Oligonucleotide Analogues with Integrated Bases and Backbone. Part 30

(2013) Lorenz Herdeis et al.   HELVETICA CHIMICA ACTA

Structure of human mitochondrial RNA polymerase elongation complex

(2013) Kathrin Schwinghammer et al.   NATURE STRUCTURAL & MOLECULAR BIOLOGY

Automated parallel synthesis of 5′-triphosphate oligonucleotides and preparation of chemically modified 5′-triphosphate small interfering RNA

(2012) Ivan Zlatev et al.   BIOORGANIC & MEDICINAL CHEMISTRY

Sensitivity of Mitochondrial Transcription and Resistance of RNA Polymerase II Dependent Nuclear Transcription to Antiviral Ribonucleosides

(2012) Jamie J. Arnold et al.   PLoS Pathogens

Unique Gene-Silencing and Structural Properties of 2′-Fluoro-Modified siRNAs

(2011) Muthiah Manoharan et al.   ANGEWANDTE CHEMIE-INTERNATIONAL EDITION

Coupled 5′ Nucleotide Recognition and Processivity in Xrn1-Mediated mRNA Decay

(2011) Martin Jinek et al.   MOLECULAR CELL

Configuration of the 5′-Methyl Group Modulates the Biophysical and Biological Properties of Locked Nucleic Acid (LNA) Oligonucleotides

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

Synthesis of Nucleoside Di- and Triphosphates and Dinucleoside Polyphosphates withcycloSal-Nucleotides

(2009) Svenja Warnecke et al.   JOURNAL OF ORGANIC CHEMISTRY

Photocleavable Initiator Nucleotide Substrates for an Aldolase Ribozyme

(2008) Stefan Fusz et al.   JOURNAL OF ORGANIC CHEMISTRY