Current prospects for RNA interference-based therapies (Q28235733)

Strategies for silencing human disease using RNA interference

Europe PubMed Central

title

Current prospects for RNA interference-based therapies (English)

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Paul B McCray

2

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author name string

Beverly L Davidson

series ordinal

1

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language of work or name

English

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publication date

May 2011

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published in

Nature Reviews Genetics

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volume

12

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page(s)

329-40

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issue

5

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cites work

1 reference

Potent and specific genetic interference by double-stranded RNA in Caenorhabditis elegans

21 January 2018

1 reference

RNA interference is mediated by 21- and 22-nucleotide RNAs

21 January 2018

1 reference

Inhibition of respiratory viruses by nasally administered siRNA.

21 January 2018

1 reference

Misinterpreting the therapeutic effects of small interfering RNA caused by immune stimulation

21 January 2018

1 reference

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

21 January 2018

1 reference

Sequence-specific potent induction of IFN-alpha by short interfering RNA in plasmacytoid dendritic cells through TLR7

21 January 2018

1 reference

Coexpressed RIG-I agonist enhances humoral immune response to influenza virus DNA vaccine

21 January 2018

1 reference

Small RNA sorting: matchmaking for Argonautes.

21 January 2018

1 reference

Small silencing RNAs: an expanding universe

21 January 2018

1 reference

Prediction of mammalian microRNA targets

21 January 2018

1 reference

Expanding the microRNA targeting code: functional sites with centered pairing

21 January 2018

1 reference

An mRNA m7G cap binding-like motif within human Ago2 represses translation

21 January 2018

1 reference

MicroRNA silencing through RISC recruitment of eIF6

21 January 2018

1 reference

Drosophila miR2 induces pseudo-polysomes and inhibits translation initiation.

21 January 2018

1 reference

P bodies: at the crossroads of post-transcriptional pathways

21 January 2018

1 reference

CCR4-NOT deadenylates mRNA associated with RNA-induced silencing complexes in human cells

21 January 2018

1 reference

Mammalian microRNAs predominantly act to decrease target mRNA levels

21 January 2018

1 reference

Relief of microRNA-mediated translational repression in human cells subjected to stress

21 January 2018

1 reference

Passenger-strand cleavage facilitates assembly of siRNA into Ago2-containing RNAi enzyme complexes

21 January 2018

1 reference

Argonaute2 cleaves the anti-guide strand of siRNA during RISC activation

21 January 2018

1 reference

Asymmetry in the assembly of the RNAi enzyme complex

21 January 2018

1 reference

Functional siRNAs and miRNAs Exhibit Strand Bias

21 January 2018

1 reference

Hierarchical rules for Argonaute loading in Drosophila

21 January 2018

1 reference

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

21 January 2018

1 reference

RNAi: double-stranded RNA directs the ATP-dependent cleavage of mRNA at 21 to 23 nucleotide intervals

21 January 2018

1 reference

Synthetic dsRNA Dicer substrates enhance RNAi potency and efficacy.

21 January 2018

1 reference

Synthetic shRNAs as potent RNAi triggers.

21 January 2018

1 reference

siRNA and innate immunity

21 January 2018

1 reference

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

21 January 2018

1 reference

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

21 January 2018

1 reference

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

21 January 2018

1 reference

In situ vaccination with a TLR9 agonist induces systemic lymphoma regression: a phase I/II study

21 January 2018

1 reference

Design of noninflammatory synthetic siRNA mediating potent gene silencing in vivo.

21 January 2018

1 reference

Potent and persistent in vivo anti-HBV activity of chemically modified siRNAs

21 January 2018

1 reference

Rational design of cationic lipids for siRNA delivery

21 January 2018

1 reference

Structural variations and stabilising modifications of synthetic siRNAs in mammalian cells

21 January 2018

1 reference

RNAi and small interfering RNAs in human disease therapeutic applications

21 January 2018

1 reference

Efficient and targeted delivery of siRNA in vivo

21 January 2018

1 reference

RNAi nanomedicines: challenges and opportunities within the immune system.

21 January 2018

1 reference

Knocking down barriers: advances in siRNA delivery

21 January 2018

1 reference

Therapeutic silencing of an endogenous gene by systemic administration of modified siRNAs

21 January 2018

1 reference

Mechanisms and optimization of in vivo delivery of lipophilic siRNAs.

21 January 2018

1 reference

Durable protection from Herpes Simplex Virus-2 transmission following intravaginal application of siRNAs targeting both a viral and host gene

21 January 2018

1 reference

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

21 January 2018

1 reference

RNAi-mediated gene silencing in non-human primates.

21 January 2018

1 reference

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

21 January 2018

1 reference

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

21 January 2018

1 reference

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

21 January 2018

1 reference

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

21 January 2018

1 reference

T cell-specific siRNA delivery suppresses HIV-1 infection in humanized mice

21 January 2018

1 reference

Systemic leukocyte-directed siRNA delivery revealing cyclin D1 as an anti-inflammatory target

21 January 2018

1 reference

RNAi-mediated CCR5 silencing by LFA-1-targeted nanoparticles prevents HIV infection in BLT mice

21 January 2018

1 reference

Transvascular delivery of small interfering RNA to the central nervous system.

21 January 2018

1 reference

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

21 January 2018

1 reference

Artificial microRNAs as siRNA shuttles: improved safety as compared to shRNAs in vitro and in vivo

21 January 2018

1 reference

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

21 January 2018

1 reference

Fatality in mice due to oversaturation of cellular microRNA/short hairpin RNA pathways

21 January 2018

1 reference

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

21 January 2018

1 reference

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

21 January 2018

1 reference

Expression of shRNA from a tissue-specific pol II promoter is an effective and safe RNAi therapeutic

21 January 2018

1 reference

Recognition and cleavage of primary microRNA precursors by the nuclear processing enzyme Drosha

21 January 2018

1 reference

Polycistronic RNA polymerase II expression vectors for RNA interference based on BIC/miR-155.

21 January 2018

1 reference

Enhanced gene silencing of HIV-1 specific siRNA using microRNA designed hairpins

21 January 2018

1 reference

Biological gene delivery vehicles: beyond viral vectors

21 January 2018

1 reference

Bacterially derived 400 nm particles for encapsulation and cancer cell targeting of chemotherapeutics

21 January 2018

1 reference

Short hairpin RNA-expressing bacteria elicit RNA interference in mammals

21 January 2018

1 reference

Cequent Pharmaceuticals, Inc.: the biological pitcher for RNAi therapeutics.

21 January 2018

1 reference

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

21 January 2018

1 reference

Viral vectors for gene transfer: current status of gene therapeutics

21 January 2018

1 reference

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

21 January 2018

1 reference

Recent advances in lentiviral vector development and applications

21 January 2018

1 reference

Receptors and entry cofactors for retroviruses include single and multiple transmembrane-spanning proteins as well as newly described glycophosphatidylinositol-anchored and secreted proteins.

21 January 2018

1 reference

HSV as a vector in vaccine development and gene therapy

21 January 2018

1 reference

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

21 January 2018

1 reference

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

21 January 2018

1 reference

Optimization of feline immunodeficiency virus vectors for RNA interference

21 January 2018

1 reference

Regulated and multiple miRNA and siRNA delivery into primary cells by a lentiviral platform

21 January 2018

1 reference

Strategies in designing multigene expression units to downregulate HIV-1.

21 January 2018

1 reference

Gene editing in human stem cells using zinc finger nucleases and integrase-defective lentiviral vector delivery

21 January 2018

1 reference

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

21 January 2018

1 reference

Genetic therapies against HIV.

21 January 2018

1 reference

The lymphocytic choriomeningitis virus envelope glycoprotein targets lentiviral gene transfer vector to neural progenitors in the murine brain

21 January 2018

1 reference

Protection against lethal influenza virus challenge by RNA interference in vivo.

21 January 2018

1 reference

Inhibition of influenza virus production in virus-infected mice by RNA interference

21 January 2018

1 reference

Inefficient cationic lipid-mediated siRNA and antisense oligonucleotide transfer to airway epithelial cells in vivo.

21 January 2018

1 reference

Epithelial sodium channel inhibition in primary human bronchial epithelia by transfected siRNA.

21 January 2018

1 reference

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

21 January 2018

1 reference

Combinatorial RNAi against HIV-1 using extended short hairpin RNAs

21 January 2018

1 reference

RNA-based gene therapy for HIV with lentiviral vector-modified CD34(+) cells in patients undergoing transplantation for AIDS-related lymphoma

21 January 2018

1 reference

Inhibition of hepatitis B virus in mice by RNA interference

21 January 2018

1 reference

Interference of hepatitis C virus RNA replication by short interfering RNAs

21 January 2018

1 reference

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

21 January 2018

1 reference

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

21 January 2018

1 reference

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

21 January 2018

1 reference

Efficient hepatitis C virus particle formation requires diacylglycerol acyltransferase-1

21 January 2018

1 reference

Polo-like kinase 1 is involved in hepatitis C virus replication by hyperphosphorylating NS5A

21 January 2018

1 reference

An siRNA-based microbicide protects mice from lethal herpes simplex virus 2 infection

21 January 2018

1 reference

RNAi suppresses polyglutamine-induced neurodegeneration in a model of spinocerebellar ataxia.

21 January 2018

1 reference

RNA interference improves motor and neuropathological abnormalities in a Huntington's disease mouse model

21 January 2018

1 reference

Intrastriatal rAAV-mediated delivery of anti-huntingtin shRNAs induces partial reversal of disease progression in R6/1 Huntington's disease transgenic mice

21 January 2018

1 reference

rAAV-mediated shRNA ameliorated neuropathology in Huntington disease model mouse

21 January 2018

1 reference

Allele-specific RNA silencing of mutant ataxin-3 mediates neuroprotection in a rat model of Machado-Joseph disease

21 January 2018

1 reference

Sustained effects of nonallele-specific Huntingtin silencing

21 January 2018

1 reference

Silencing mutant SOD1 using RNAi protects against neurodegeneration and extends survival in an ALS model

21 January 2018

1 reference

Lentiviral-mediated silencing of SOD1 through RNA interference retards disease onset and progression in a mouse model of ALS.

21 January 2018

1 reference

Processing of pre-microRNAs by the Dicer-1-Loquacious complex in Drosophila cells

21 January 2018

1 reference

Silencing of human alpha-synuclein in vitro and in rat brain using lentiviral-mediated RNAi

21 January 2018

1 reference

Targeting BACE1 with siRNAs ameliorates Alzheimer disease neuropathology in a transgenic model

21 January 2018

1 reference

Herpes simplex virus RNAi and neprilysin gene transfer vectors reduce accumulation of Alzheimer's disease-related amyloid-beta peptide in vivo

21 January 2018

1 reference

Eight years of clinical improvement in MPTP-lesioned primates after gene therapy with AAV2-hAADC.

21 January 2018

1 reference

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

21 January 2018

1 reference

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

21 January 2018

1 reference

Silencing primary dystonia: lentiviral-mediated RNA interference therapy for DYT1 dystonia.

21 January 2018

1 reference

Nonallele-specific silencing of mutant and wild-type huntingtin demonstrates therapeutic efficacy in Huntington's disease mice.

21 January 2018

1 reference

Five siRNAs targeting three SNPs may provide therapy for three-quarters of Huntington's disease patients

21 January 2018

1 reference

CAG expansion in the Huntington disease gene is associated with a specific and targetable predisposing haplogroup

21 January 2018

1 reference

siRNA-mediated gene silencing in vitro and in vivo.

21 January 2018

1 reference

RNA interference in adult mice.

21 January 2018

1 reference

Targeted RNA interference for hepatic fibrosis

21 January 2018

1 reference

BI 2536, a potent and selective inhibitor of polo-like kinase 1, inhibits tumor growth in vivo.

21 January 2018

1 reference

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

21 January 2018

1 reference

Exploiting and antagonizing microRNA regulation for therapeutic and experimental applications

21 January 2018

1 reference

Interferon modulation of cellular microRNAs as an antiviral mechanism

21 January 2018

1 reference

Silencing of microRNAs in vivo with 'antagomirs'

21 January 2018

1 reference

Therapeutic silencing of microRNA-122 in primates with chronic hepatitis C virus infection

21 January 2018

1 reference

MicroRNA sponges: competitive inhibitors of small RNAs in mammalian cells

21 January 2018

1 reference

Cutaneous short-interfering RNA therapy.

21 January 2018

1 reference

Improved retinal function in a mouse model of dominant retinitis pigmentosa following AAV-delivered gene therapy

21 January 2018

1 reference

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

21 January 2018

1 reference

MicroRNAs add a new dimension to cardiovascular disease

21 January 2018

1 reference

Inhibition of ABCB1 (MDR1) expression by an siRNA nanoparticulate delivery system to overcome drug resistance in osteosarcoma

21 January 2018

1 reference

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

21 January 2018

1 reference

Targeting Stat3 in the myeloid compartment drastically improves the in vivo antitumor functions of adoptively transferred T cells

21 January 2018

1 reference

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

21 January 2018

1 reference

Expression profiling reveals off-target gene regulation by RNAi

21 January 2018

1 reference

Widespread siRNA "off-target" transcript silencing mediated by seed region sequence complementarity

21 January 2018

1 reference

Comparison of siRNA-induced off-target RNA and protein effects

21 January 2018

1 reference

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

21 January 2018

1 reference

Small RNAs with imperfect match to endogenous mRNA repress translation. Implications for off-target activity of small inhibitory RNA in mammalian cells

21 January 2018

1 reference

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

21 January 2018

1 reference

Position-specific chemical modification of siRNAs reduces "off-target" transcript silencing

21 January 2018

1 reference

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

21 January 2018

1 reference

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

21 January 2018

1 reference

RNA polymerase III transcribes human microRNAs

21 January 2018

1 reference

Structure and activity of putative intronic miRNA promoters

21 January 2018

1 reference

Chromatin structure analyses identify miRNA promoters

21 January 2018

1 reference

The nuclear RNase III Drosha initiates microRNA processing

21 January 2018

1 reference

The Microprocessor complex mediates the genesis of microRNAs

21 January 2018

1 reference

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

21 January 2018

1 reference

Nuclear export of microRNA precursors

21 January 2018

1 reference

Exportin-5 mediates the nuclear export of pre-microRNAs and short hairpin RNAs

21 January 2018

1 reference

Ribonuclease activity and RNA binding of recombinant human Dicer

21 January 2018

1 reference

Normal microRNA maturation and germ-line stem cell maintenance requires Loquacious, a double-stranded RNA-binding domain protein

21 January 2018

1 reference

Role for a bidentate ribonuclease in the initiation step of RNA interference

21 January 2018

1 reference

ATP requirements and small interfering RNA structure in the RNA interference pathway

21 January 2018

1 reference

Sequence-dependent stimulation of the mammalian innate immune response by synthetic siRNA

21 January 2018

1 reference

The widespread regulation of microRNA biogenesis, function and decay

31 March 2020

1 reference

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

31 March 2020

1 reference

Second-generation shRNA libraries covering the mouse and human genomes

31 March 2020

1 reference

A Hybrid Vector for Ligand-Directed Tumor Targeting and Molecular Imaging

31 March 2020

1 reference

Recombinant adeno-associated virus type 2, 4, and 5 vectors: transduction of variant cell types and regions in the mammalian central nervous system.

31 March 2020

1 reference

Using siRNA in prophylactic and therapeutic regimens against SARS coronavirus in Rhesus macaque

31 March 2020

1 reference

Polyethylenimine-based siRNA nanocomplexes reprogram tumor-associated dendritic cells via TLR5 to elicit therapeutic antitumor immunity

31 March 2020

1 reference

PubMed

https://pubmed.ncbi.nlm.nih.gov/21499294

12 December 2020

inferred from PubMed ID database lookup

Lentivirus-mediated RNA interference of vascular endothelial growth factor in monkey eyes with iris neovascularization.

1 reference

PubMed

https://pubmed.ncbi.nlm.nih.gov/21499294

12 December 2020

inferred from PubMed ID database lookup

Blocking neuropilin-2 enhances corneal allograft survival by selectively inhibiting lymphangiogenesis on vascularized beds.

1 reference

PubMed

https://pubmed.ncbi.nlm.nih.gov/21499294

12 December 2020

inferred from PubMed ID database lookup

Identifiers

DOI

10.1038/NRG2968

1 reference

Consolidated OpenCitations Corpus – April 2017

Dimensions Publication ID

489319

1048606089

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Fatcat ID

release_vt7xft3wczgvpl6dyt2yjbzrwu

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Fatcat

https://api.fatcat.wiki/v0/release/vt7xft3wczgvpl6dyt2yjbzrwu

24 November 2022

mapped directly with Wikidata item

489319

1 reference

Consolidated OpenCitations Corpus – April 2017

Consolidated OpenCitations Corpus – April 2017

1 reference