Recombinant AAV as a platform for translating the therapeutic potential of RNA interference. (Q37696045)

https://www.ebi.ac.uk/europepmc/webservices/rest/search?query=EXT_ID:24352214%20AND%20SRC:MED&resulttype=core&format=json

3 March 2020

title

Recombinant AAV as a platform for translating the therapeutic potential of RNA interference (English)

1 reference

https://www.ebi.ac.uk/europepmc/webservices/rest/search?query=EXT_ID:24352214%20AND%20SRC:MED&resulttype=core&format=json

3 March 2020

1 reference

1

1 reference

https://www.ebi.ac.uk/europepmc/webservices/rest/search?query=EXT_ID:24352214%20AND%20SRC:MED&resulttype=core&format=json

3 March 2020

author name string

Mark A Kay

series ordinal

2

1 reference

https://www.ebi.ac.uk/europepmc/webservices/rest/search?query=EXT_ID:24352214%20AND%20SRC:MED&resulttype=core&format=json

3 March 2020

Christian Mueller

series ordinal

3

1 reference

https://www.ebi.ac.uk/europepmc/webservices/rest/search?query=EXT_ID:24352214%20AND%20SRC:MED&resulttype=core&format=json

3 March 2020

language of work or name

1 reference

19 December 2013

1 reference

https://www.ebi.ac.uk/europepmc/webservices/rest/search?query=EXT_ID:24352214%20AND%20SRC:MED&resulttype=core&format=json

3 March 2020

1 reference

https://www.ebi.ac.uk/europepmc/webservices/rest/search?query=EXT_ID:24352214%20AND%20SRC:MED&resulttype=core&format=json

3 March 2020

volume

22

1 reference

https://www.ebi.ac.uk/europepmc/webservices/rest/search?query=EXT_ID:24352214%20AND%20SRC:MED&resulttype=core&format=json

3 March 2020

issue

4

1 reference

https://www.ebi.ac.uk/europepmc/webservices/rest/search?query=EXT_ID:24352214%20AND%20SRC:MED&resulttype=core&format=json

3 March 2020

page(s)

692-701

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https://www.ebi.ac.uk/europepmc/webservices/rest/search?query=EXT_ID:24352214%20AND%20SRC:MED&resulttype=core&format=json

Preclinical evaluation of an anti-HCV miRNA cluster for treatment of HCV infection

3 March 2020

cites work

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The loop position of shRNAs and pre-miRNAs is critical for the accuracy of dicer processing in vivo

23 August 2017

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Embedding siRNA sequences targeting apolipoprotein B100 in shRNA and miRNA scaffolds results in differential processing and in vivo efficacy

23 August 2017

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Safe, long-term hepatic expression of anti-HCV shRNA in a nonhuman primate model

23 August 2017

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Efficacy and long-term safety of alipogene tiparvovec (AAV1-LPLS447X) gene therapy for lipoprotein lipase deficiency: an open-label trial

23 August 2017

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Integration frequency and intermolecular recombination of rAAV vectors in non-human primate skeletal muscle and liver

23 August 2017

1 reference

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Long-term, efficient inhibition of microRNA function in mice using rAAV vectors

23 August 2017

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Prostate-targeted radiosensitization via aptamer-shRNA chimeras in human tumor xenografts

23 August 2017

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Cardiac gene transfer of short hairpin RNA directed against phospholamban effectively knocks down gene expression but causes cellular toxicity in canines

23 August 2017

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MicroRNA-regulated, systemically delivered rAAV9: a step closer to CNS-restricted transgene expression

23 August 2017

1 reference

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Design of small molecule-responsive microRNAs based on structural requirements for Drosha processing

23 August 2017

1 reference

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

23 August 2017

1 reference

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Target RNA-directed trimming and tailing of small silencing RNAs

23 August 2017

1 reference

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Emerging paradigms of regulated microRNA processing

23 August 2017

1 reference

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Cellular toxicity following application of adeno-associated viral vector-mediated RNA interference in the nervous system

23 August 2017

1 reference

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

23 August 2017

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MicroRNA-122 inhibits tumorigenic properties of hepatocellular carcinoma cells and sensitizes these cells to sorafenib

23 August 2017

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Vision 1 year after gene therapy for Leber's congenital amaurosis

23 August 2017

1 reference

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Dose optimization for long-term rAAV-mediated RNA interference in the nigrostriatal projection neurons

23 August 2017

1 reference

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Therapeutic microRNA delivery suppresses tumorigenesis in a murine liver cancer model

23 August 2017

1 reference

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MicroRNA-122, a tumor suppressor microRNA that regulates intrahepatic metastasis of hepatocellular carcinoma

23 August 2017

1 reference

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

23 August 2017

1 reference

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

23 August 2017

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=3982504

miR-122 targets an anti-apoptotic gene, Bcl-w, in human hepatocellular carcinoma cell lines

23 August 2017

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=3982504

Frequency and spectrum of genomic integration of recombinant adeno-associated virus serotype 8 vector in neonatal mouse liver

23 August 2017

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=3982504

Coexpression of Argonaute-2 enhances RNA interference toward perfect match binding sites

23 August 2017

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=3982504

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

23 August 2017

1 reference

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Genomic analysis of human microRNA transcripts

23 August 2017

1 reference

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MicroRNA sponges: competitive inhibitors of small RNAs in mammalian cells

23 August 2017

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Combinatorial delivery of small interfering RNAs reduces RNAi efficacy by selective incorporation into RISC

23 August 2017

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Cyclin G1 is a target of miR-122a, a microRNA frequently down-regulated in human hepatocellular carcinoma

23 August 2017

1 reference

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Genome-wide resources of endoribonuclease-prepared short interfering RNAs for specific loss-of-function studies

23 August 2017

1 reference

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RNA polymerase III transcribes human microRNAs

23 August 2017

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

23 August 2017

1 reference

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Endogenous microRNA regulation suppresses transgene expression in hematopoietic lineages and enables stable gene transfer

23 August 2017

1 reference

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miR-122 regulation of lipid metabolism revealed by in vivo antisense targeting

23 August 2017

1 reference

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Combinatorial microRNA target predictions

23 August 2017

1 reference

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

23 August 2017

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=3982504

Conserved seed pairing, often flanked by adenosines, indicates that thousands of human genes are microRNA targets

23 August 2017

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=3982504

Introduction of a Chimeric Chalcone Synthase Gene into Petunia Results in Reversible Co-Suppression of Homologous Genes in trans

23 August 2017

1 reference

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Duplexes of 21-nucleotide RNAs mediate RNA interference in cultured mammalian cells

23 August 2017

1 reference

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

23 August 2017

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=3982504

The C. elegans heterochronic gene lin-4 encodes small RNAs with antisense complementarity to lin-14

23 August 2017

1 reference

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Structure and transcription of a human gene for H1 RNA, the RNA component of human RNase P.

23 August 2017

1 reference

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Diversifying microRNA sequence and function

23 August 2017

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A largely random AAV integration profile after LPLD gene therapy

6 September 2017

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Managing microRNAs with vector-encoded decoy-type inhibitors

6 September 2017

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Antiviral strategies combining antiretroviral drugs with RNAi-mediated attack on HIV-1 and cellular co-factors

6 September 2017

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Dicer-independent processing of short hairpin RNAs

6 September 2017

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Potent microRNA suppression by RNA Pol II-transcribed 'Tough Decoy' inhibitors

6 September 2017

1 reference

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In vivo gene delivery by nonviral vectors: overcoming hurdles?

6 September 2017

1 reference

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Generation of hairpin-based RNAi vectors for biological and therapeutic application.

6 September 2017

1 reference

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The dose can make the poison: lessons learned from adverse in vivo toxicities caused by RNAi overexpression

6 September 2017

1 reference

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Rational design of therapeutic siRNAs: minimizing off-targeting potential to improve the safety of RNAi therapy for Huntington's disease.

14 September 2017

1 reference

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

14 September 2017

1 reference

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Artificial control of gene expression in mammalian cells by modulating RNA interference through aptamer-small molecule interaction

14 September 2017

1 reference

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Defining the RNA polymerase III transcriptome: Genome-wide localization of the RNA polymerase III transcription machinery in human cells

14 September 2017

1 reference

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In vivo knock-down of multidrug resistance transporters ABCC1 and ABCC2 by AAV-delivered shRNAs and by artificial miRNAs.

28 September 2017

1 reference

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Lethal toxicity caused by expression of shRNA in the mouse striatum: implications for therapeutic design.

23 June 2018

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=3982504

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

23 June 2018

1 reference

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Mir-142-3p target sequences reduce transgene-directed immunogenicity following intramuscular adeno-associated virus 1 vector-mediated gene delivery

23 June 2018

1 reference

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Long-term rescue of retinal structure and function by rhodopsin RNA replacement with a single adeno-associated viral vector in P23H RHO transgenic mice.

1 September 2018

1 reference

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Sustained miRNA-mediated knockdown of mutant AAT with simultaneous augmentation of wild-type AAT has minimal effect on global liver miRNA profiles.

1 September 2018

1 reference

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An α-synuclein AAV gene silencing vector ameliorates a behavioral deficit in a rat model of Parkinson's disease, but displays toxicity in dopamine neurons

1 September 2018

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=3982504

microRNA122-regulated transgene expression increases specificity of cardiac gene transfer upon intravenous delivery of AAV9 vectors

1 September 2018

1 reference

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

1 September 2018

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=3982504

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

1 September 2018

1 reference

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Endogenous microRNA can be broadly exploited to regulate transgene expression according to tissue, lineage and differentiation state

1 September 2018

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=3982504

Small interfering RNAs mediate sequence-independent gene suppression and induce immune activation by signaling through toll-like receptor 3.

1 September 2018

1 reference

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Sustained high-level expression of human factor IX (hFIX) after liver-targeted delivery of recombinant adeno-associated virus encoding the hFIX gene in rhesus macaques

1 September 2018

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=3982504

RNA interference in adult mice.

1 September 2018

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=3982504

Flavonoid genes in petunia: addition of a limited number of gene copies may lead to a suppression of gene expression

1 September 2018

1 reference

PubMed

https://pubmed.ncbi.nlm.nih.gov/24352214

inferred from PubMed ID database lookup

12 December 2020

based on heuristic

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

1 reference

PubMed

https://pubmed.ncbi.nlm.nih.gov/24352214

inferred from PubMed ID database lookup

12 December 2020

based on heuristic

Case 15-2009: a man with coma after cardiac arrest

1 reference

PubMed

https://pubmed.ncbi.nlm.nih.gov/24352214

inferred from PubMed ID database lookup

12 December 2020

based on heuristic

Identifiers

DOI

10.1038/MT.2013.285

1 reference

https://www.ebi.ac.uk/europepmc/webservices/rest/search?query=EXT_ID:24352214%20AND%20SRC:MED&resulttype=core&format=json

3 March 2020

1 reference

https://www.ebi.ac.uk/europepmc/webservices/rest/search?query=EXT_ID:24352214%20AND%20SRC:MED&resulttype=core&format=json

3 March 2020

1 reference

https://www.ebi.ac.uk/europepmc/webservices/rest/search?query=EXT_ID:24352214%20AND%20SRC:MED&resulttype=core&format=json