Reversal of siRNA-mediated gene silencing in vivo

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

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

Selection of GalNAc-conjugated siRNAs with limited off-target-driven rat hepatotoxicity

(2018) Maja M. Janas et al.   Nature Communications

Safety, tolerability, and antiviral effect of RG-101 in patients with chronic hepatitis C: a phase 1B, double-blind, randomised controlled trial

(2017) Meike H van der Ree et al.   LANCET

Clinical Proof of Concept for a Novel Hepatocyte-Targeting GalNAc-siRNA Conjugate

(2017) Tracy S. Zimmermann et al.   MOLECULAR THERAPY

A Highly Durable RNAi Therapeutic Inhibitor of PCSK9

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

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

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

siRNA carrying an (E)-vinylphosphonate moiety at the 5΄ end of the guide strand augments gene silencing by enhanced binding to human Argonaute-2

(2016) Elad Elkayam et al.   NUCLEIC ACIDS RESEARCH

Hepatocyte-Specific Delivery of siRNAs Conjugated to Novel Non-nucleosidic TrivalentN-Acetylgalactosamine Elicits Robust Gene Silencing in Vivo

(2015) Kallanthottathil G. Rajeev et al.   CHEMBIOCHEM

Reversing Antisense Oligonucleotide Activity with a Sense Oligonucleotide Antidote: Proof of Concept Targeting Prothrombin

(2015) Jeff R. Crosby 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

Structural basis for microRNA targeting

(2014) N. T. Schirle et al.   SCIENCE

featureCounts: an efficient general purpose program for assigning sequence reads to genomic features

(2013) Y. Liao et al.   BIOINFORMATICS

Effect of an RNA interference drug on the synthesis of proprotein convertase subtilisin/kexin type 9 (PCSK9) and the concentration of serum LDL cholesterol in healthy volunteers: a randomised, single-blind, placebo-controlled, phase 1 trial

(2013) Kevin Fitzgerald et al.   LANCET

Safety and Efficacy of RNAi Therapy for Transthyretin Amyloidosis

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

Treatment of HCV Infection by Targeting MicroRNA

(2013) Harry L.A. Janssen et al.   NEW ENGLAND JOURNAL OF MEDICINE

Pharmacological Inhibition of a MicroRNA Family in Nonhuman Primates by a Seed-Targeting 8-Mer AntimiR

(2013) V. Rottiers et al.   Science Translational Medicine

STAR: ultrafast universal RNA-seq aligner

(2012) Alexander Dobin et al.   BIOINFORMATICS

Silencing of microRNA families by seed-targeting tiny LNAs

(2011) Susanna Obad et al.   NATURE GENETICS

Quantitative evaluation of siRNA delivery in vivo

(2010) Y. Pei et al.   RNA