(Q36321469)

13 August 2017

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title

Adeno-associated virus-based gene therapy for inherited disorders (English)

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13 August 2017

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Terence R Flotte

1

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PubMed ID

16306183

13 August 2017

language of work or name

English

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publication date

1 December 2005

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13 August 2017

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13 August 2017

58

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13 August 2017

6

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13 August 2017

1143-1147

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https://scigraph.springernature.com/pub.10.1203/01.pdr.0000189226.03684.fe

13 August 2017

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cites work

A phase I study of an adeno-associated virus-CFTR gene vector in adult CF patients with mild lung disease

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https://api.crossref.org/works/10.1203%2F01.PDR.0000189226.03684.FE

Adeno-associated virus vectors for gene therapy of cystic fibrosis

21 January 2018

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Safety and biological efficacy of an adeno-associated virus vector-cystic fibrosis transmembrane regulator (AAV-CFTR) in the cystic fibrosis maxillary sinus.

21 January 2018

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https://api.crossref.org/works/10.1203%2F01.PDR.0000189226.03684.FE

Phase I trial of intranasal and endobronchial administration of a recombinant adeno-associated virus serotype 2 (rAAV2)-CFTR vector in adult cystic fibrosis patients: a two-part clinical study

21 January 2018

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https://api.crossref.org/works/10.1203%2F01.PDR.0000189226.03684.FE

A phase II, double-blind, randomized, placebo-controlled clinical trial of tgAAVCF using maxillary sinus delivery in patients with cystic fibrosis with antrostomies.

21 January 2018

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https://api.crossref.org/works/10.1203%2F01.PDR.0000189226.03684.FE

Phase I trial of intramuscular injection of a recombinant adeno-associated virus alpha 1-antitrypsin (rAAV2-CB-hAAT) gene vector to AAT-deficient adults

21 January 2018

1 reference

https://api.crossref.org/works/10.1203%2F01.PDR.0000189226.03684.FE

Repeated adeno-associated virus serotype 2 aerosol-mediated cystic fibrosis transmembrane regulator gene transfer to the lungs of patients with cystic fibrosis: a multicenter, double-blind, placebo-controlled trial

21 January 2018

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https://api.crossref.org/works/10.1203%2F01.PDR.0000189226.03684.FE

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

21 January 2018

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Clades of Adeno-associated viruses are widely disseminated in human tissues.

21 January 2018

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https://api.crossref.org/works/10.1203%2F01.PDR.0000189226.03684.FE

The cryptic life style of adeno-associated virus

21 January 2018

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https://api.crossref.org/works/10.1203%2F01.PDR.0000189226.03684.FE

Organization of adeno-associated virus DNA in latently infected Detroit 6 cells

21 January 2018

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https://api.crossref.org/works/10.1203%2F01.PDR.0000189226.03684.FE

Site-specific integration by adeno-associated virus

21 January 2018

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https://api.crossref.org/works/10.1203%2F01.PDR.0000189226.03684.FE

Mapping and direct visualization of a region-specific viral DNA integration site on chromosome 19q13-qter

21 January 2018

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Adeno-associated virus: integration at a specific chromosomal locus

21 January 2018

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A Human Parvovirus, Adeno-Associated Virus, as a Eucaryotic Vector: Transient Expression and Encapsidation of the Procaryotic Gene for Chloramphenicol Acetyltransferase

21 January 2018

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Adeno-associated virus vector for high-frequency integration, expression, and rescue of genes in mammalian cells

21 January 2018

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Use of adeno-associated virus as a mammalian DNA cloning vector: transduction of neomycin resistance into mammalian tissue culture cells

21 January 2018

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Recombinant adeno-associated virus purification using novel methods improves infectious titer and yield

21 January 2018

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Gene expression from adeno-associated virus vectors in airway epithelial cells

21 January 2018

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Defective regulation of outwardly rectifying Cl- channels by protein kinase A corrected by insertion of CFTR

21 January 2018

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Stable in vivo expression of the cystic fibrosis transmembrane conductance regulator with an adeno-associated virus vector

21 January 2018

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Adeno-associated virus vector gene expression occurs in nondividing cells in the absence of vector DNA integration

21 January 2018

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AlphaVbeta5 integrin: a co-receptor for adeno-associated virus type 2 infection

21 January 2018

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Distinct classes of proteasome-modulating agents cooperatively augment recombinant adeno-associated virus type 2 and type 5-mediated transduction from the apical surfaces of human airway epithelia

21 January 2018

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Inhibition of recombinant adeno-associated virus (rAAV) transduction by bronchial secretions from cystic fibrosis patients

21 January 2018

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The alpha 1-antitrypsin gene and its deficiency states

21 January 2018

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Gene delivery to skeletal muscle results in sustained expression and systemic delivery of a therapeutic protein

21 January 2018

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Recombinant adeno-associated virus for muscle directed gene therapy

21 January 2018

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

21 January 2018

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Effect of DNA-dependent protein kinase on the molecular fate of the rAAV2 genome in skeletal muscle

21 January 2018

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https://api.crossref.org/works/10.1203%2F01.PDR.0000189226.03684.FE

DNA-dependent PK inhibits adeno-associated virus DNA integration

21 January 2018

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https://api.crossref.org/works/10.1203%2F01.PDR.0000189226.03684.FE

Several log increase in therapeutic transgene delivery by distinct adeno-associated viral serotype vectors

21 January 2018

1 reference

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10.1203/01.PDR.0000189226.03684.FE

21 January 2018

Identifiers

DOI

1 reference

PubMed ID

16306183

Dimensions Publication ID

13 August 2017

0 references

1 reference

PubMed ID

16306183