(Q40115238)

17 September 2017

In vivo post-transcriptional gene silencing of alpha-1 antitrypsin by adeno-associated virus vectors expressing siRNA. (English)

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17 September 2017

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Martha Campbell-Thompson

8

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17 September 2017

Jeffrey H Teckman

10

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author name string

Pedro E Cruz

1

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17 September 2017

Christian Mueller

2

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17 September 2017

Travis L Cossette

3

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17 September 2017

Alexandra Golant

4

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17 September 2017

Qiushi Tang

5

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17 September 2017

Stuart G Beattie

6

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17 September 2017

Mark Brantly

7

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17 September 2017

Keith S Blomenkamp

9

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17 September 2017

Terence R Flotte

11

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17 September 2017

25 June 2007

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17 September 2017

Laboratory Investigation

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17 September 2017

87

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17 September 2017

9

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17 September 2017

893-902

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https://scigraph.springernature.com/pub.10.1038/labinvest.3700629

17 September 2017

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cites work

Loop-sheet polymerization: the structural basis of Z alpha 1-antitrypsin accumulation in the liver

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21 January 2018

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The alpha 1-antitrypsin gene and its deficiency states

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7 January 2021

Alpha 1-antitrypsin deficiency, emphysema, and liver disease. Genetic basis and strategies for therapy

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7 January 2021

α 1 ‐antitrypsin deficiency and liver disease

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7 January 2021

Alpha-1-antitrypsin deficiency: biochemistry and clinical manifestations

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7 January 2021

Histopathology of α1-antitrypsin liver disease in a transgenic mouse model

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7 January 2021

Effect of the Z mutation on the physical and inhibitory properties of alpha 1-antitrypsin

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7 January 2021

Loop-sheet polymerization: the mechanism of alpha1-antitrypsin deficiency

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7 January 2021

Alpha1-antitrypsin polymerization and the serpinopathies: pathobiology and prospects for therapy

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7 January 2021

Hypersensitive mousetraps, α1-antitrypsin deficiency and dementia

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7 January 2021

Alpha1-antitrypsin deficiency. 4: Molecular pathophysiology

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7 January 2021

Polymerisation underlies alpha1-antitrypsin deficiency, dementia and other serpinopathies

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7 January 2021

Alpha1-antitrypsin deficiency--a model for conformational diseases

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7 January 2021

Sustained secretion of human alpha-1-antitrypsin from murine muscle transduced with adeno-associated virus vectors

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7 January 2021

Stable therapeutic serum levels of human alpha-1 antitrypsin (AAT) after portal vein injection of recombinant adeno-associated virus (rAAV) vectors

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7 January 2021

Intramuscular administration of recombinant adeno-associated virus 2 alpha-1 antitrypsin (rAAV-SERPINA1) vectors in a nonhuman primate model: safety and immunologic aspects

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7 January 2021

Efficient hepatic delivery and expression from a recombinant adeno-associated virus 8 pseudotyped alpha1-antitrypsin vector

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7 January 2021

Therapeutic level of functional human alpha 1 antitrypsin (hAAT) secreted from murine muscle transduced by adeno-associated virus (rAAV1) vector

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7 January 2021

Immune responses to recombinant adeno-associated virus vectors: putting preclinical findings into perspective

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7 January 2021

Phase I trial of intramuscular injection of a recombinant adeno-associated virus serotype 2 alphal-antitrypsin (AAT) vector in AAT-deficient adults

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7 January 2021

A novel SV40-based vector successfully transduces and expresses an alpha 1-antitrypsin ribozyme in a human hepatoma-derived cell line

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7 January 2021

In vivo selection of hepatocytes transduced with adeno-associated viral vectors.

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7 January 2021

New recombinant serotypes of AAV vectors.

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7 January 2021

High-level transgene expression in nonhuman primate liver with novel adeno-associated virus serotypes containing self-complementary genomes

1 reference

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7 January 2021

Liver transduction with recombinant adeno-associated virus is primarily restricted by capsid serotype not vector genotype

1 reference

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7 January 2021

Functional anatomy of siRNAs for mediating efficient RNAi in Drosophila melanogaster embryo lysate

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7 January 2021

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

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7 January 2021

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

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7 January 2021

Production and purification of serotype 1, 2, and 5 recombinant adeno-associated viral vectors

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7 January 2021

Targeting recombinant adeno-associated virus vectors to enhance gene transfer to pancreatic islets and liver

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7 January 2021

Multiple tissues express alpha 1-antitrypsin in transgenic mice and man.

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7 January 2021

Accumulation of PiZ alpha 1-antitrypsin causes liver damage in transgenic mice

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7 January 2021

Neonatal hepatitis induced by alpha 1-antitrypsin: a transgenic mouse model

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7 January 2021

Quantitative isolation of alphalAT mutant Z protein polymers from human and mouse livers and the effect of heat

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7 January 2021

Analyses of hepatocellular proliferation in a mouse model of alpha-1-antitrypsin deficiency

1 reference

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7 January 2021

Indomethacin increases liver damage in a murine model of liver injury from alpha-1-antitrypsin deficiency

1 reference

https://api.crossref.org/works/10.1038%2FLABINVEST.3700629

7 January 2021

Novel adeno-associated viruses from rhesus monkeys as vectors for human gene therapy

1 reference

https://api.crossref.org/works/10.1038%2FLABINVEST.3700629

7 January 2021

Preclinical in vivo evaluation of pseudotyped adeno-associated virus vectors for liver gene therapy

1 reference

https://api.crossref.org/works/10.1038%2FLABINVEST.3700629

7 January 2021

Nonrandom transduction of recombinant adeno-associated virus vectors in mouse hepatocytes in vivo: cell cycling does not influence hepatocyte transduction

1 reference

https://api.crossref.org/works/10.1038%2FLABINVEST.3700629

7 January 2021

A limited number of transducible hepatocytes restricts a wide-range linear vector dose response in recombinant adeno-associated virus-mediated liver transduction

1 reference

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7 January 2021

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

1 reference

https://api.crossref.org/works/10.1038%2FLABINVEST.3700629

7 January 2021

10.1038/LABINVEST.3700629

Identifiers

DOI

1 reference

17 September 2017

1 reference