Therapeutic application of RNAi: is mRNA targeting finally ready for prime time? (Q24669624)

Journal of Clinical Investigation

Europe PubMed Central

title

Therapeutic application of RNAi: is mRNA targeting finally ready for prime time? (English)

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1

Dirk Grimm

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author name string

Mark A Kay

series ordinal

2

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language of work or name

English

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publication date

December 2007

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published in

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volume

117

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issue

12

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page(s)

3633-41

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cites work

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Identification of eight members of the Argonaute family in the human genome

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The genetic engineering of hematopoietic stem cells: the rise of lentiviral vectors, the conundrum of the ltr, and the promise of lineage-restricted vectors

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RNAi in combination with a ribozyme and TAR decoy for treatment of HIV infection in hematopoietic cell gene therapy

28 September 2017

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RNAi therapeutics: a potential new class of pharmaceutical drugs

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Designing siRNA that distinguish between genes that differ by a single nucleotide

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28 September 2017

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

28 September 2017

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Off-target effects by siRNA can induce toxic phenotype

28 September 2017

1 reference

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

28 September 2017

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3' UTR seed matches, but not overall identity, are associated with RNAi off-targets

28 September 2017

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RNA interference in cancer

28 September 2017

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Therapeutic short hairpin RNA expression in the liver: viral targets and vectors

28 September 2017

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Prospects of RNA interference therapy for cancer

28 September 2017

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28 September 2017

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28 September 2017

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28 September 2017

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

28 September 2017

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Suppression of RNA interference by adenovirus virus-associated RNA.

28 September 2017

1 reference

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In vivo RNA interference-mediated ablation of MDR1 P-glycoprotein

28 September 2017

1 reference

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The silent revolution: RNA interference as basic biology, research tool, and therapeutic

28 September 2017

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Adeno-associated virus vectors for short hairpin RNA expression

28 September 2017

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=2096424

Enhancement of hypoxia-induced tumor cell death in vitro and radiation therapy in vivo by use of small interfering RNA targeted to hypoxia-inducible factor-1alpha

28 September 2017

1 reference

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Unlocking the potential of the human genome with RNA interference

28 September 2017

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Small interfering RNA-induced transcriptional gene silencing in human cells

28 September 2017

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Efficient gene transfer of HIV-1-specific short hairpin RNA into human lymphocytic cells using recombinant adeno-associated virus vectors

28 September 2017

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=2096424

From virus evolution to vector revolution: use of naturally occurring serotypes of adeno-associated virus (AAV) as novel vectors for human gene therapy

28 September 2017

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=2096424

Small interfering RNA (siRNA) targeting VEGF effectively inhibits ocular neovascularization in a mouse model.

28 September 2017

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=2096424

Independent combinatorial effect of antisense oligonucleotides and RNAi-mediated specific inhibition of the recombinant rat P2X3 receptor

28 September 2017

1 reference

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Self-inactivating lentivirus vector for safe and efficient in vivo gene delivery

28 September 2017

1 reference

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

28 September 2017

1 reference

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Evidence for gene transfer and expression of factor IX in haemophilia B patients treated with an AAV vector.

30 May 2018

1 reference

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Mechanisms and optimization of in vivo delivery of lipophilic siRNAs.

30 May 2018

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Immune responses to AAV capsid: are mice not humans after all?

28 November 2018

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Exploring the uses of RNAi--gene knockdown and the Nobel Prize.

28 November 2018

1 reference

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RNAi-mediated gene silencing in non-human primates.

28 November 2018

1 reference

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LNA-modified oligonucleotides mediate specific inhibition of microRNA function.

28 November 2018

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MicroRNA therapeutics: a new niche for antisense nucleic acids.

28 November 2018

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A genetic strategy to treat sickle cell anemia by coregulating globin transgene expression and RNA interference

28 November 2018

1 reference

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Design of noninflammatory synthetic siRNA mediating potent gene silencing in vivo

28 November 2018

1 reference

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Suppression of ocular neovascularization with siRNA targeting VEGF receptor 1.

28 November 2018

1 reference

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Effective inhibition of cell growth and invasion of melanoma by combined suppression of BRAF (V599E) and Skp2 with lentiviral RNAi.

28 November 2018

1 reference

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Inhibition of respiratory viruses by nasally administered siRNA.

28 November 2018

1 reference

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Inhibition of respiratory syncytial virus infection with intranasal siRNA nanoparticles targeting the viral NS1 gene

28 November 2018

1 reference

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

28 November 2018

1 reference

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Adeno-associated virus terminal repeat (TR) mutant generates self-complementary vectors to overcome the rate-limiting step to transduction in vivo

28 November 2018

1 reference

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Induction of an interferon response by RNAi vectors in mammalian cells

28 November 2018

1 reference

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RNA interference in adult mice.

28 November 2018

1 reference

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28 November 2018

Gene therapy on trial

1 reference

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Different delivery methods-different expression profiles

28 November 2018

1 reference

12 December 2020

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

1 reference

12 December 2020

Antibody mediated in vivo delivery of small interfering RNAs via cell-surface receptors

1 reference

12 December 2020

Noise amidst the silence: off-target effects of siRNAs?

1 reference

12 December 2020

A microRNA-regulated lentiviral vector mediates stable correction of hemophilia B mice

1 reference

Crossref

https://api.crossref.org/works/10.1172%2FJCI34129

7 January 2021

inferred from DOI database lookup

Identifiers

DOI

10.1172/JCI34129

0 references

Fatcat ID

release_fuczompozveonkjrsbykygteyi

1 reference

Fatcat

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

24 November 2022