Gene therapy progress and prospects: recombinant adeno-associated virus (rAAV) vectors (Q35712206)

9 August 2017

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title

Gene therapy progress and prospects: recombinant adeno-associated virus (rAAV) vectors (English)

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9 August 2017

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Flotte TR

1

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9 August 2017

publication date

1 May 2004

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9 August 2017

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9 August 2017

volume

11

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9 August 2017

issue

10

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9 August 2017

page(s)

805-810

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Novel transcriptional regulatory signals in the adeno-associated virus terminal repeat A/D junction element

9 August 2017

cites work

1 reference

https://api.crossref.org/works/10.1038%2FSJ.GT.3302233

Adeno-associated viruses undergo substantial evolution in primates during natural infections

21 January 2018

1 reference

https://api.crossref.org/works/10.1038%2FSJ.GT.3302233

The atomic structure of adeno-associated virus (AAV-2), a vector for human gene therapy.

21 January 2018

1 reference

https://api.crossref.org/works/10.1038%2FSJ.GT.3302233

Identification of amino acid residues in the capsid proteins of adeno-associated virus type 2 that contribute to heparan sulfate proteoglycan binding

21 January 2018

1 reference

https://api.crossref.org/works/10.1038%2FSJ.GT.3302233

Mutational analysis of the adeno-associated virus type 2 (AAV2) capsid gene and construction of AAV2 vectors with altered tropism

21 January 2018

1 reference

https://api.crossref.org/works/10.1038%2FSJ.GT.3302233

Adeno-associated virus type 5 (AAV5) but not AAV2 binds to the apical surfaces of airway epithelia and facilitates gene transfer

21 January 2018

1 reference

https://api.crossref.org/works/10.1038%2FSJ.GT.3302233

Adeno-associated virus serotype 4 (AAV4) and AAV5 both require sialic acid binding for hemagglutination and efficient transduction but differ in sialic acid linkage specificity

21 January 2018

1 reference

https://api.crossref.org/works/10.1038%2FSJ.GT.3302233

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

21 January 2018

1 reference

https://api.crossref.org/works/10.1038%2FSJ.GT.3302233

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

21 January 2018

1 reference

https://api.crossref.org/works/10.1038%2FSJ.GT.3302233

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

21 January 2018

1 reference

https://api.crossref.org/works/10.1038%2FSJ.GT.3302233

Receptor targeting of adeno-associated virus vectors

21 January 2018

1 reference

https://api.crossref.org/works/10.1038%2FSJ.GT.3302233

Cross-packaging of a single adeno-associated virus (AAV) type 2 vector genome into multiple AAV serotypes enables transduction with broad specificity

21 January 2018

1 reference

https://api.crossref.org/works/10.1038%2FSJ.GT.3302233

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

21 January 2018

1 reference

https://api.crossref.org/works/10.1038%2FSJ.GT.3302233

Endosomal processing limits gene transfer to polarized airway epithelia by adeno-associated virus.

21 January 2018

1 reference

https://api.crossref.org/works/10.1038%2FSJ.GT.3302233

Impaired intracellular trafficking of adeno-associated virus type 2 vectors limits efficient transduction of murine fibroblasts

21 January 2018

1 reference

https://api.crossref.org/works/10.1038%2FSJ.GT.3302233

Ubiquitination of both adeno-associated virus type 2 and 5 capsid proteins affects the transduction efficiency of recombinant vectors.

21 January 2018

1 reference

https://api.crossref.org/works/10.1038%2FSJ.GT.3302233

Infectious entry pathway of adeno-associated virus and adeno-associated virus vectors

21 January 2018

1 reference

https://api.crossref.org/works/10.1038%2FSJ.GT.3302233

Adeno-associated virus type 2-mediated gene transfer: role of cellular FKBP52 protein in transgene expression

21 January 2018

1 reference

https://api.crossref.org/works/10.1038%2FSJ.GT.3302233

Self-complementary recombinant adeno-associated virus (scAAV) vectors promote efficient transduction independently of DNA synthesis

21 January 2018

1 reference

https://api.crossref.org/works/10.1038%2FSJ.GT.3302233

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

21 January 2018

1 reference

https://api.crossref.org/works/10.1038%2FSJ.GT.3302233

Recombinant AAV-mediated gene delivery using dual vector heterodimerization.

21 January 2018

1 reference

https://api.crossref.org/works/10.1038%2FSJ.GT.3302233

Overcoming adeno-associated virus vector size limitation through viral DNA heterodimerization

21 January 2018

1 reference

https://api.crossref.org/works/10.1038%2FSJ.GT.3302233

Efficient trans-splicing in the retina expands the utility of adeno-associated virus as a vector for gene therapy.

21 January 2018

1 reference

https://api.crossref.org/works/10.1038%2FSJ.GT.3302233

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

21 January 2018

1 reference

https://api.crossref.org/works/10.1038%2FSJ.GT.3302233

Streamlined large-scale production of recombinant adeno-associated virus (rAAV) vectors

21 January 2018

1 reference

https://api.crossref.org/works/10.1038%2FSJ.GT.3302233

Purification of recombinant adeno-associated virus vectors by column chromatography and its performance in vivo

21 January 2018

1 reference

https://api.crossref.org/works/10.1038%2FSJ.GT.3302233

Gene therapy for pyruvate dehydrogenase E1alpha deficiency using recombinant adeno-associated virus 2 (rAAV2) vectors

21 January 2018

1 reference

https://api.crossref.org/works/10.1038%2FSJ.GT.3302233

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

21 January 2018

1 reference

https://api.crossref.org/works/10.1038%2FSJ.GT.3302233

Extrachromosomal recombinant adeno-associated virus vector genomes are primarily responsible for stable liver transduction in vivo

21 January 2018

1 reference

https://api.crossref.org/works/10.1038%2FSJ.GT.3302233

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

21 January 2018

1 reference

https://api.crossref.org/works/10.1038%2FSJ.GT.3302233

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

21 January 2018

1 reference

https://api.crossref.org/works/10.1038%2FSJ.GT.3302233

CMV-beta-actin promoter directs higher expression from an adeno-associated viral vector in the liver than the cytomegalovirus or elongation factor 1 alpha promoter and results in therapeutic levels of human factor X in mice

21 January 2018

1 reference

https://api.crossref.org/works/10.1038%2FSJ.GT.3302233

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

1 reference

https://api.crossref.org/works/10.1038%2FSJ.GT.3302233

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

1 reference

https://api.crossref.org/works/10.1038%2FSJ.GT.3302233

A phase I study of aerosolized administration of tgAAVCF to cystic fibrosis subjects with mild lung disease

21 January 2018

1 reference

https://api.crossref.org/works/10.1038%2FSJ.GT.3302233

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

21 January 2018

1 reference

https://api.crossref.org/works/10.1038%2FSJ.GT.3302233

AAV-mediated factor IX gene transfer to skeletal muscle in patients with severe hemophilia B.

21 January 2018

1 reference

https://api.crossref.org/works/10.1038%2FSJ.GT.3302233

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

21 January 2018

1 reference

https://api.crossref.org/works/10.1038%2FSJ.GT.3302233

Bronchoalveolar fluid is not a major hindrance to virus-mediated gene therapy in cystic fibrosis.

21 January 2018

1 reference

https://api.crossref.org/works/10.1038%2FSJ.GT.3302233

Improved method of recombinant AAV2 delivery for systemic targeted gene therapy

21 January 2018

1 reference

https://api.crossref.org/works/10.1038%2FSJ.GT.3302233

Repeat transduction in the mouse lung by using adeno-associated virus vectors with different serotypes

21 January 2018

1 reference

https://api.crossref.org/works/10.1038%2FSJ.GT.3302233

Correction of a genetic defect by nuclear transplantation and combined cell and gene therapy

21 January 2018

1 reference

https://api.crossref.org/works/10.1038%2FSJ.GT.3302233

Obstacles to human hematopoietic stem cell transduction by recombinant adeno-associated virus 2 vectors.

21 January 2018

1 reference

https://api.crossref.org/works/10.1038%2FSJ.GT.3302233

inferred from DOI database lookup

7 January 2021

based on heuristic

Deposition and expression of aerosolized rAAV vectors in the lungs of Rhesus macaques

1 reference

https://api.crossref.org/works/10.1038%2FSJ.GT.3302233

inferred from DOI database lookup

7 January 2021

based on heuristic

Identifiers

DOI

10.1038/SJ.GT.3302233

1 reference

9 August 2017

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