Knocking down disease: a progress report on siRNA therapeutics

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

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

Protease-triggered siRNA delivery vehicles

(2015) David B. Rozema et al.   JOURNAL OF CONTROLLED RELEASE

Dysregulation of microRNA biogenesis and gene silencing in cancer

(2015) Akiko Hata et al.   Science Signaling

Dysregulation of microRNA biogenesis and gene silencing in cancer

(2015) Akiko Hata et al.   Science Signaling

First-in-Human Phase I Study of the Liposomal RNA Interference Therapeutic Atu027 in Patients With Advanced Solid Tumors

(2014) Beate Schultheis et al.   JOURNAL OF CLINICAL ONCOLOGY

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

Systemic Delivery of a miR34a Mimic as a Potential Therapeutic for Liver Cancer

(2014) C. L. Daige et al.   MOLECULAR CANCER THERAPEUTICS

Efficient delivery of RNAi prodrugs containing reversible charge-neutralizing phosphotriester backbone modifications

(2014) Bryan R Meade et al.   NATURE BIOTECHNOLOGY

Genome editing with Cas9 in adult mice corrects a disease mutation and phenotype

(2014) Hao Yin et al.   NATURE BIOTECHNOLOGY

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

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

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

Antisense oligonucleotides: modifications and clinical trials

(2014) Vivek K. Sharma et al.   MedChemComm

The mTOR pathway in hepatic malignancies

(2013) Mamatha Bhat et al.   HEPATOLOGY

Aptamer-targeted inhibition of mTOR in T cells enhances antitumor immunity

(2013) Alexey Berezhnoy et al.   JOURNAL OF CLINICAL INVESTIGATION

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

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

Biodegradable Lipids Enabling Rapidly Eliminated Lipid Nanoparticles for Systemic Delivery of RNAi Therapeutics

(2013) Martin A Maier et al.   MOLECULAR THERAPY

Hepatocyte-targeted RNAi Therapeutics for the Treatment of Chronic Hepatitis B Virus Infection

(2013) Christine I Wooddell et al.   MOLECULAR THERAPY

Knockdown of β-catenin with Dicer-Substrate siRNAs Reduces Liver Tumor Burden In vivo

(2013) Henryk Dudek et al.   MOLECULAR THERAPY

Efficient and specific gene knockdown by small interfering RNAs produced in bacteria

(2013) Linfeng Huang et al.   NATURE BIOTECHNOLOGY

Image-based analysis of lipid nanoparticle–mediated siRNA delivery, intracellular trafficking and endosomal escape

(2013) Jerome Gilleron et al.   NATURE BIOTECHNOLOGY

Delivery materials for siRNA therapeutics

(2013) Rosemary Kanasty et al.   NATURE MATERIALS

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

First-in-Humans Trial of an RNA Interference Therapeutic Targeting VEGF and KSP in Cancer Patients with Liver Involvement

(2013) Josep Tabernero et al.   Cancer Discovery

Toxicity profiling of several common RNAi-based nanomedicines: a comparative study

(2013) Dalit Landesman-Milo et al.   Drug Delivery and Translational Research

Harnessing a Physiologic Mechanism for siRNA Delivery With Mimetic Lipoprotein Particles

(2012) Tomoko Nakayama et al.   MOLECULAR THERAPY

Galectin 8 targets damaged vesicles for autophagy to defend cells against bacterial invasion

(2012) Teresa L. M. Thurston et al.   NATURE

Targeted Delivery of PLK1-siRNA by ScFv Suppresses Her2+ Breast Cancer Growth and Metastasis

(2012) Y.-d. Yao et al.   Science Translational Medicine

Inhibition of HIV transmission in human cervicovaginal explants and humanized mice using CD4 aptamer-siRNA chimeras

(2011) Lee Adam Wheeler et al.   JOURNAL OF CLINICAL INVESTIGATION

Biodistribution of Small Interfering RNA at the Organ and Cellular Levels after Lipid Nanoparticle-mediated Delivery

(2011) Bin Shi et al.   JOURNAL OF HISTOCHEMISTRY & CYTOCHEMISTRY

Delivery of siRNA to the mouse brain by systemic injection of targeted exosomes

(2011) Lydia Alvarez-Erviti et al.   NATURE BIOTECHNOLOGY

Systemic Administration of PRO051 in Duchenne's Muscular Dystrophy

(2011) Nathalie M. Goemans et al.   NEW ENGLAND JOURNAL OF MEDICINE

RNA Targeting Therapeutics: Molecular Mechanisms of Antisense Oligonucleotides as a Therapeutic Platform

(2010) C. Frank Bennett et al.   Annual Review of Pharmacology and Toxicology

The systemic toxicity of positively charged lipid nanoparticles and the role of Toll-like receptor 4 in immune activation

(2010) Ranit Kedmi et al.   BIOMATERIALS

Highly Efficient Reprogramming to Pluripotency and Directed Differentiation of Human Cells with Synthetic Modified mRNA

(2010) Luigi Warren et al.   Cell Stem Cell

Targeting Polo-like Kinase in Cancer Therapy

(2010) Y. Degenhardt et al.   CLINICAL CANCER RESEARCH

Postexposure protection of non-human primates against a lethal Ebola virus challenge with RNA interference: a proof-of-concept study

(2010) Thomas W Geisbert et al.   LANCET

Mipomersen, an apolipoprotein B synthesis inhibitor, for lowering of LDL cholesterol concentrations in patients with homozygous familial hypercholesterolaemia: a randomised, double-blind, placebo-controlled trial

(2010) Frederick J Raal et al.   LANCET

Targeted Delivery of RNAi Therapeutics With Endogenous and Exogenous Ligand-Based Mechanisms

(2010) Akin Akinc et al.   MOLECULAR THERAPY

Evidence of RNAi in humans from systemically administered siRNA via targeted nanoparticles

(2010) Mark E. Davis et al.   NATURE

Rational design of cationic lipids for siRNA delivery

(2010) Sean C Semple et al.   NATURE BIOTECHNOLOGY

Durable Protection from Herpes Simplex Virus-2 Transmission Following Intravaginal Application of siRNAs Targeting Both a Viral and Host Gene

(2009) Yichao Wu et al.   Cell Host & Microbe

Confirming the RNAi-mediated mechanism of action of siRNA-based cancer therapeutics in mice

(2009) Adam D. Judge et al.   JOURNAL OF CLINICAL INVESTIGATION

In vivo delivery of siRNA to immune cells by conjugation to a TLR9 agonist enhances antitumor immune responses

(2009) Marcin Kortylewski et al.   NATURE BIOTECHNOLOGY

T Cell-Specific siRNA Delivery Suppresses HIV-1 Infection in Humanized Mice

(2008) Priti Kumar et al.   CELL

Sequence- and target-independent angiogenesis suppression by siRNA via TLR3

(2008) Mark E. Kleinman et al.   NATURE

A combinatorial library of lipid-like materials for delivery of RNAi therapeutics

(2008) Akin Akinc et al.   NATURE BIOTECHNOLOGY

5′-triphosphate-siRNA: turning gene silencing and Rig-I activation against melanoma

(2008) Hendrik Poeck et al.   NATURE MEDICINE

Systemic Leukocyte-Directed siRNA Delivery Revealing Cyclin D1 as an Anti-Inflammatory Target

(2008) D. Peer et al.   SCIENCE