Comparison of partially and fully chemically-modified siRNA in conjugate-mediated delivery in vivo

The chemical evolution of oligonucleotide therapies of clinical utility

(2017) Anastasia Khvorova et al.   NATURE BIOTECHNOLOGY

Identification of metabolically stable 5΄-phosphate analogs that support single-stranded siRNA activity

(2017) Thazha P. Prakash et al.   NUCLEIC ACIDS RESEARCH

5΄-Vinylphosphonate improves tissue accumulation and efficacy of conjugated siRNAs in vivo

(2017) Reka A. Haraszti et al.   NUCLEIC ACIDS RESEARCH

Impact of enhanced metabolic stability on pharmacokinetics and pharmacodynamics of GalNAc–siRNA conjugates

(2017) Jayaprakash K. Nair et al.   NUCLEIC ACIDS RESEARCH

Pharmacokinetic Profiling of Conjugated Therapeutic Oligonucleotides: A High-Throughput Method Based Upon Serial Blood Microsampling Coupled to Peptide Nucleic Acid Hybridization Assay

(2017) Bruno M.D.C. Godinho et al.   Nucleic Acid Therapeutics

Synergistic effect of phosphorothioate, 5′-vinylphosphonate and GalNAc modifications for enhancing activity of synthetic siRNA

(2016) Thazha P. Prakash et al.   BIOORGANIC & MEDICINAL CHEMISTRY LETTERS

5′-(E)-Vinylphosphonate: A Stable Phosphate Mimic Can Improve the RNAi Activity of siRNA-GalNAc Conjugates

(2016) Rubina Parmar et al.   CHEMBIOCHEM

Structural Analysis of Human Argonaute-2 Bound to a Modified siRNA Guide

(2016) Nicole T. Schirle et al.   JOURNAL OF THE AMERICAN CHEMICAL SOCIETY

Visualization of self-delivering hydrophobically modified siRNA cellular internalization

(2016) Socheata Ly et al.   NUCLEIC ACIDS RESEARCH

A High-Throughput Method for Direct Detection of Therapeutic Oligonucleotide-Induced Gene SilencingIn Vivo

(2016) Andrew H. Coles et al.   Nucleic Acid Therapeutics

Silencing Myostatin Using Cholesterol-conjugated siRNAs Induces Muscle Growth

(2016) Tayeba Khan et al.   Molecular Therapy-Nucleic Acids

Docosahexaenoic Acid Conjugation Enhances Distribution and Safety of siRNA upon Local Administration in Mouse Brain

(2016) Mehran Nikan et al.   Molecular Therapy-Nucleic Acids

Single-Molecule Imaging Reveals that Argonaute Reshapes the Binding Properties of Its Nucleic Acid Guides

(2015) William E. Salomon et al.   CELL

Guanabenz (Wytensin™) selectively enhances uptake and efficacy of hydrophobically modified siRNAs

(2015) Maire F. Osborn et al.   NUCLEIC ACIDS RESEARCH

Hydrophobically Modified siRNAs Silence Huntingtin mRNA in Primary Neurons and Mouse Brain

(2015) Julia F Alterman et al.   Molecular Therapy-Nucleic Acids

The 5′ Binding MID Domain of Human Argonaute2 Tolerates Chemically Modified Nucleotide Analogues

(2015) Glen F. Deleavey et al.   Nucleic Acid Therapeutics

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

In vivo endothelial siRNA delivery using polymeric nanoparticles with low molecular weight

(2014) James E. Dahlman et al.   Nature Nanotechnology

Systematic evaluation of antibody-mediated siRNA delivery using an industrial platform of THIOMAB–siRNA conjugates

(2014) Trinna L. Cuellar et al.   NUCLEIC ACIDS RESEARCH

Structural basis for microRNA targeting

(2014) N. T. Schirle et al.   SCIENCE

Phosphorothioates, Essential Components of Therapeutic Oligonucleotides

(2014) Fritz Eckstein   Nucleic Acid Therapeutics

Novel Hydrophobically Modified Asymmetric RNAi Compounds (sd-rxRNA) Demonstrate Robust Efficacy in the Eye

(2013) Michael Byrne et al.   JOURNAL OF OCULAR PHARMACOLOGY AND THERAPEUTICS

Argonaute Divides Its RNA Guide into Domains with Distinct Functions and RNA-Binding Properties

(2012) Liang Meng Wee et al.   CELL

Single-Stranded RNAs Use RNAi to Potently and Allele-Selectively Inhibit Mutant Huntingtin Expression

(2012) Dongbo Yu et al.   CELL

Single-Stranded siRNAs Activate RNAi in Animals

(2012) Walt F. Lima et al.   CELL

Defined Folate-PEG-siRNA Conjugates for Receptor-specific Gene Silencing

(2012) Christian Dohmen et al.   Molecular Therapy-Nucleic Acids

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

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

Silencing disease genes in the laboratory and the clinic

(2011) Jonathan K Watts et al.   JOURNAL OF PATHOLOGY

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

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

Transfection Techniques for Neuronal Cells

(2010) D. Karra et al.   JOURNAL OF NEUROSCIENCE

Synergistic effects between analogs of DNA and RNA improve the potency of siRNA-mediated gene silencing

(2010) Glen F. Deleavey et al.   NUCLEIC ACIDS RESEARCH

Chemically modified siRNA: tools and applications

(2008) J WATTS et al.   DRUG DISCOVERY TODAY

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

(2008) Kazutaka Nishina et al.   MOLECULAR THERAPY