Current prospects for RNA interference-based therapies

Unity and diversity in the human adenoviruses: exploiting alternative entry pathways for gene therapy

(2015) Kathryn Hall et al.   BIOCHEMICAL JOURNAL

Therapeutic in vivo gene transfer for genetic disease using AAV: progress and challenges

(2011) Federico Mingozzi et al.   NATURE REVIEWS GENETICS

Highly potent and specific siRNAs against E6 or E7 genes of HPV16- or HPV18-infected cervical cancers

(2010) J T-C Chang et al.   CANCER GENE THERAPY

Targeting Stat3 in the Myeloid Compartment Drastically Improves the In vivo Antitumor Functions of Adoptively Transferred T Cells

(2010) A. Herrmann et al.   CANCER RESEARCH

Cardiac-targeted delivery of regulatory RNA molecules and genes for the treatment of heart failure

(2010) W. Poller et al.   CARDIOVASCULAR RESEARCH

MicroRNAs Add a New Dimension to Cardiovascular Disease

(2010) Eric M. Small et al.   CIRCULATION

Efficient and targeted delivery of siRNA in vivo

(2010) Min Suk Shim et al.   FEBS Journal

Computational and experimental identification of mirtrons in Drosophila melanogaster and Caenorhabditis elegans

(2010) W.-J. Chung et al.   GENOME RESEARCH

Inhibition of hepatitis C virus replication using adeno-associated virus vector delivery of an exogenous anti-hepatitis C virus microrna cluster

(2010) Xiao Yang et al.   HEPATOLOGY

Argonaute proteins are key determinants of RNAi efficacy, toxicity, and persistence in the adult mouse liver

(2010) Dirk Grimm et al.   JOURNAL OF CLINICAL INVESTIGATION

In Situ Vaccination With a TLR9 Agonist Induces Systemic Lymphoma Regression: A Phase I/II Study

(2010) Joshua D. Brody et al.   JOURNAL OF CLINICAL ONCOLOGY

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

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

Induction of therapeutic gene silencing in leukocyte-implicated diseases by targeted and stabilized nanoparticles: A mini-review

(2010) Dan Peer   JOURNAL OF CONTROLLED RELEASE

Coexpressed RIG-I Agonist Enhances Humoral Immune Response to Influenza Virus DNA Vaccine

(2010) J. M. Luke et al.   JOURNAL OF VIROLOGY

Polo-Like Kinase 1 Is Involved in Hepatitis C Virus Replication by Hyperphosphorylating NS5A

(2010) Y.-C. Chen et al.   JOURNAL OF VIROLOGY

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

CCR4-NOT Deadenylates mRNA Associated with RNA-Induced Silencing Complexes in Human Cells

(2010) X. Piao et al.   MOLECULAR AND CELLULAR BIOLOGY

Expanding the MicroRNA Targeting Code: Functional Sites with Centered Pairing

(2010) Chanseok Shin et al.   MOLECULAR CELL

Eight Years of Clinical Improvement in MPTP-Lesioned Primates After Gene Therapy With AAV2-hAADC

(2010) Piotr Hadaczek et al.   MOLECULAR THERAPY

Recent Advances in Lentiviral Vector Development and Applications

(2010) Janka Mátrai et al.   MOLECULAR THERAPY

RNAi nanomedicines: challenges and opportunities within the immune system

(2010) Shiri Weinstein et al.   NANOTECHNOLOGY

Mammalian microRNAs predominantly act to decrease target mRNA levels

(2010) Huili Guo et al.   NATURE

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

Efficient hepatitis C virus particle formation requires diacylglycerol acyltransferase-1

(2010) Eva Herker et al.   NATURE MEDICINE

Specific gene transfer to neurons, endothelial cells and hematopoietic progenitors with lentiviral vectors

(2010) Brigitte Anliker et al.   NATURE METHODS

Small RNA sorting: matchmaking for Argonautes

(2010) Benjamin Czech et al.   NATURE REVIEWS GENETICS

A screen of chemical modifications identifies position-specific modification by UNA to most potently reduce siRNA off-target effects

(2010) Jesper B. Bramsen et al.   NUCLEIC ACIDS RESEARCH

A role for human Dicer in pre-RISC loading of siRNAs

(2010) Kumi Sakurai et al.   NUCLEIC ACIDS RESEARCH

Inhibition of ABCB1 (MDR1) Expression by an siRNA Nanoparticulate Delivery System to Overcome Drug Resistance in Osteosarcoma

(2010) Michiro Susa et al.   PLoS One

A randomized, double-blind, placebo-controlled study of an RNAi-based therapy directed against respiratory syncytial virus

(2010) J. DeVincenzo et al.   PROCEEDINGS OF THE NATIONAL ACADEMY OF SCIENCES OF THE UNITED STATES OF AMERICA

Structure and activity of putative intronic miRNA promoters

(2010) A. M. Monteys et al.   RNA

Reduced seed region-based off-target activity with lentivirus-mediated RNAi

(2010) R. A. Klinghoffer et al.   RNA

Titers of lentiviral vectors encoding shRNAs and miRNAs are reduced by different mechanisms that require distinct repair strategies

(2010) Y. P. Liu et al.   RNA

RNA-Based Gene Therapy for HIV with Lentiviral Vector-Modified CD34+ Cells in Patients Undergoing Transplantation for AIDS-Related Lymphoma

(2010) D. L. DiGiusto et al.   Science Translational Medicine

RNAi and small interfering RNAs in human disease therapeutic applications

(2010) Monica R. Lares et al.   TRENDS IN BIOTECHNOLOGY

CAG Expansion in the Huntington Disease Gene Is Associated with a Specific and Targetable Predisposing Haplogroup

(2009) Simon C. Warby et al.   AMERICAN JOURNAL OF HUMAN GENETICS

Sustained effects of nonallele-specificHuntingtinsilencing

(2009) Valérie Drouet et al.   ANNALS OF NEUROLOGY

A novel in vivo siRNA delivery system specifically targeting dendritic cells and silencing CD40 genes for immunomodulation

(2009) X. Zheng et al.   BLOOD

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

Five siRNAs Targeting Three SNPs May Provide Therapy for Three-Quarters of Huntington's Disease Patients

(2009) Edith L. Pfister et al.   CURRENT BIOLOGY

Targeted RNA interference for hepatic fibrosis

(2009) Ping-Fang Hu et al.   EXPERT OPINION ON BIOLOGICAL THERAPY

Cutaneous short-interfering RNA therapy

(2009) B Geusens et al.   Expert Opinion on Drug Delivery

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

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

Novel biodegradable lipid nano complex for siRNA delivery significantly improving the chemosensitivity of human colon cancer stem cells to paclitaxel

(2009) Changxing Liu et al.   JOURNAL OF CONTROLLED RELEASE

Hierarchical Rules for Argonaute Loading in Drosophila

(2009) Benjamin Czech et al.   MOLECULAR CELL

Improved Retinal Function in a Mouse Model of Dominant Retinitis Pigmentosa Following AAV-delivered Gene Therapy

(2009) Naomi Chadderton et al.   MOLECULAR THERAPY

Combinatorial RNAi Against HIV-1 Using Extended Short Hairpin RNAs

(2009) Ying Poi Liu et al.   MOLECULAR THERAPY

Regulated and Multiple miRNA and siRNA Delivery Into Primary Cells by a Lentiviral Platform

(2009) Mario Amendola et al.   MOLECULAR THERAPY

Nonallele-specific Silencing of Mutant and Wild-type Huntingtin Demonstrates Therapeutic Efficacy in Huntington's Disease Mice

(2009) Ryan L Boudreau et al.   MOLECULAR THERAPY

Biological Gene Delivery Vehicles: Beyond Viral Vectors

(2009) Yiqi Seow et al.   MOLECULAR THERAPY

RNAi-mediated CCR5 Silencing by LFA-1-targeted Nanoparticles Prevents HIV Infection in BLT Mice

(2009) Sang-Soo Kim et al.   MOLECULAR THERAPY

Sequential treatment of drug-resistant tumors with targeted minicells containing siRNA or a cytotoxic drug

(2009) Jennifer A MacDiarmid et al.   NATURE BIOTECHNOLOGY

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

Systemic administration of optimized aptamer-siRNA chimeras promotes regression of PSMA-expressing tumors

(2009) Justin P Dassie et al.   NATURE BIOTECHNOLOGY

Molecular signatures of disease brain endothelia provide new sites for CNS-directed enzyme therapy

(2009) Yong Hong Chen et al.   NATURE MEDICINE

Knocking down barriers: advances in siRNA delivery

(2009) Kathryn A. Whitehead et al.   NATURE REVIEWS DRUG DISCOVERY

Small silencing RNAs: an expanding universe

(2009) Megha Ghildiyal et al.   NATURE REVIEWS GENETICS

Exploiting and antagonizing microRNA regulation for therapeutic and experimental applications

(2009) Brian D. Brown et al.   NATURE REVIEWS GENETICS

Efficient silencing of gene expression with modular trimeric Pol II expression cassettes comprising microRNA shuttles

(2009) A. Ely et al.   NUCLEIC ACIDS RESEARCH

Selection, characterization and application of new RNA HIV gp 120 aptamers for facile delivery of Dicer substrate siRNAs into HIV infected cells

(2009) Jiehua Zhou et al.   NUCLEIC ACIDS RESEARCH

siRNA and Innate Immunity

(2009) Marjorie Robbins et al.   OLIGONUCLEOTIDES

Small interfering RNA-induced TLR3 activation inhibits blood and lymphatic vessel growth

(2009) W. G. Cho et al.   PROCEEDINGS OF THE NATIONAL ACADEMY OF SCIENCES OF THE UNITED STATES OF AMERICA

Optimization of linear double-stranded RNA for the production of multiple siRNAs targeting hepatitis C virus

(2009) D. Shin et al.   RNA

Emerging roles of microRNAs as molecular switches in the integrated circuit of the cancer cell

(2009) G. Sotiropoulou et al.   RNA

MicroRNA-like off-target transcript regulation by siRNAs is species specific

(2009) J. Burchard et al.   RNA

Therapeutic Silencing of MicroRNA-122 in Primates with Chronic Hepatitis C Virus Infection

(2009) R. E. Lanford et al.   SCIENCE

Epithelial Sodium Channel Inhibition in Primary Human Bronchial Epithelia by Transfected siRNA

(2008) Emanuela Caci et al.   AMERICAN JOURNAL OF RESPIRATORY CELL AND MOLECULAR BIOLOGY

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

(2008) Priti Kumar et al.   CELL

Chromatin structure analyses identify miRNA promoters

(2008) F. Ozsolak et al.   GENES & DEVELOPMENT

Misinterpreting the Therapeutic Effects of Small Interfering RNA Caused by Immune Stimulation

(2008) Marjorie Robbins et al.   HUMAN GENE THERAPY

Expression of shRNA From a Tissue-specific pol II Promoter Is an Effective and Safe RNAi Therapeutic

(2008) Jeffery C Giering et al.   MOLECULAR THERAPY

Artificial MicroRNAs as siRNA Shuttles: Improved Safety as Compared to shRNAs In vitro and In vivo

(2008) Ryan L Boudreau et al.   MOLECULAR THERAPY

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

(2008) Mark E. Kleinman et al.   NATURE

Allele-Specific RNA Silencing of Mutant Ataxin-3 Mediates Neuroprotection in a Rat Model of Machado-Joseph Disease

(2008) Sandro Alves et al.   PLoS One

Artificial miRNAs mitigate shRNA-mediated toxicity in the brain: Implications for the therapeutic development of RNAi

(2008) J. L. McBride et al.   PROCEEDINGS OF THE NATIONAL ACADEMY OF SCIENCES OF THE UNITED STATES OF AMERICA

Therapeutic RNAi targeting PCSK9 acutely lowers plasma cholesterol in rodents and LDL cholesterol in nonhuman primates

(2008) M. Frank-Kamenetsky et al.   PROCEEDINGS OF THE NATIONAL ACADEMY OF SCIENCES OF THE UNITED STATES OF AMERICA

Minimizing variables among hairpin-based RNAi vectors reveals the potency of shRNAs

(2008) R. L. Boudreau et al.   RNA

Experimental validation of the importance of seed complement frequency to siRNA specificity

(2008) E. M. Anderson et al.   RNA

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

(2008) D. Peer et al.   SCIENCE