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

The chemical evolution of oligonucleotide therapies of clinical utility

(2017) Anastasia Khvorova et al.   NATURE BIOTECHNOLOGY

Oligonucleotide Therapeutics — A New Class of Cholesterol-Lowering Drugs

(2017) Anastasia Khvorova   NEW ENGLAND JOURNAL OF MEDICINE

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

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

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Alnylam terminates revusiran program, stock plunges

(2016) Ken Garber   NATURE BIOTECHNOLOGY

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

Enhancing Endosomal Escape for Intracellular Delivery of Macromolecular Biologic Therapeutics

(2016) Peter Lönn et al.   Scientific Reports

siRNA Conjugates Carrying Sequentially Assembled Trivalent N-Acetylgalactosamine Linked Through Nucleosides Elicit Robust Gene Silencing In Vivo in Hepatocytes

(2015) Shigeo Matsuda et al.   ACS Chemical Biology

Cellular 5′-3′ mRNA Exonuclease Xrn1 Controls Double-Stranded RNA Accumulation and Anti-Viral Responses

(2015) Hannah M. Burgess et al.   Cell Host & Microbe

Out of one mutation, many Huntington's disease effects

(2015) Roger L Albin   LANCET NEUROLOGY

Treating the whole body in Huntington's disease

(2015) Jeffrey B Carroll et al.   LANCET NEUROLOGY

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

(2015) Alfica Sehgal et al.   NATURE MEDICINE

2′-Fluoro-modified phosphorothioate oligonucleotide can cause rapid degradation of P54nrb and PSF

(2015) Wen Shen 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

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

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

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

Liver as a target for oligonucleotide therapeutics

(2013) Alfica Sehgal et al.   JOURNAL OF HEPATOLOGY

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

Safety and Efficacy of RNAi Therapy for Transthyretin Amyloidosis

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

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

MicroRNA turnover: when, how, and why

(2012) Stefan Rüegger et al.   TRENDS IN BIOCHEMICAL SCIENCES

Target-Mediated Protection of Endogenous MicroRNAs in C. elegans

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Structural basis for 5′-nucleotide base-specific recognition of guide RNA by human AGO2

(2010) Filipp Frank et al.   NATURE

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

Differential regulation of microRNA stability

(2010) S. Bail et al.   RNA

Active turnover modulates mature microRNA activity in Caenorhabditis elegans

(2009) Saibal Chatterjee et al.   NATURE

Analysis of acyclic nucleoside modifications in siRNAs finds sensitivity at position 1 that is restored by 5′-terminal phosphorylation both in vitro and in vivo

(2009) Denise M. Kenski et al.   NUCLEIC ACIDS RESEARCH