Sustained correction of glycogen storage disease type II using adeno-associated virus serotype 1 vectors (Q45457856)

14 December 2017

title

Sustained correction of glycogen storage disease type II using adeno-associated virus serotype 1 vectors (English)

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14 December 2017

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glycogen storage disease

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Mah C

1

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14 December 2017

Cresawn KO

2

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14 December 2017

Fraites TJ Jr

3

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14 December 2017

Pacak CA

4

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14 December 2017

Lewis MA

5

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14 December 2017

Zolotukhin I

6

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14 December 2017

Byrne BJ

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14 December 2017

publication date

1 September 2005

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14 December 2017

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14 December 2017

volume

12

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14 December 2017

issue

18

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14 December 2017

page(s)

1405-1409

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alpha-Glucosidase deficiency in generalized glycogenstorage disease (Pompe's disease).

14 December 2017

cites work

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https://api.crossref.org/works/10.1038/SJ.GT.3302550

Acid alpha-glucosidase deficiency (glycogenosis type II, Pompe disease).

21 January 2018

1 reference

https://api.crossref.org/works/10.1038/SJ.GT.3302550

Glycogenosis type II (acid maltase deficiency)

21 January 2018

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https://api.crossref.org/works/10.1038/SJ.GT.3302550

21 January 2018

https://api.crossref.org/works/10.1038/SJ.GT.3302550

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7 January 2021

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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

1 reference

https://api.crossref.org/works/10.1038/SJ.GT.3302550

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

21 January 2018

1 reference

https://api.crossref.org/works/10.1038/SJ.GT.3302550

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/SJ.GT.3302550

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

1 reference

https://api.crossref.org/works/10.1038/SJ.GT.3302550

Sustained and complete phenotype correction of hemophilia B mice following intramuscular injection of AAV1 serotype vectors

21 January 2018

1 reference

https://api.crossref.org/works/10.1038/SJ.GT.3302550

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

21 January 2018

1 reference

https://api.crossref.org/works/10.1038/SJ.GT.3302550

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

21 January 2018

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https://api.crossref.org/works/10.1038/SJ.GT.3302550

Systemic delivery of genes to striated muscles using adeno-associated viral vectors.

21 January 2018

1 reference

https://api.crossref.org/works/10.1038/SJ.GT.3302550

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/SJ.GT.3302550

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/SJ.GT.3302550

Characterization of tissue tropism determinants of adeno-associated virus type 1.

21 January 2018

1 reference

https://api.crossref.org/works/10.1038/SJ.GT.3302550

Adeno-associated virus-mediated transfer of human acid maltase gene results in a transient reduction of glycogen accumulation in muscle of Japanese quail with acid maltase deficiency

21 January 2018

1 reference

https://api.crossref.org/works/10.1038/SJ.GT.3302550

Rescue of enzyme deficiency in embryonic diaphragm in a mouse model of metabolic myopathy: Pompe disease

21 January 2018

1 reference

https://api.crossref.org/works/10.1038/SJ.GT.3302550

Packaging of an AAV vector encoding human acid α-glucosidase for gene therapy in glycogen storage disease type II with a modified hybrid adenovirus-AAV vector

21 January 2018

1 reference

https://api.crossref.org/works/10.1038/SJ.GT.3302550

Targeted disruption of the acid alpha-glucosidase gene in mice causes an illness with critical features of both infantile and adult human glycogen storage disease type II

21 January 2018

1 reference

https://api.crossref.org/works/10.1038/SJ.GT.3302550

Correction of the enzymatic and functional deficits in a model of Pompe disease using adeno-associated virus vectors

21 January 2018

1 reference

https://api.crossref.org/works/10.1038/SJ.GT.3302550

Efficient transduction of skeletal muscle using vectors based on adeno-associated virus serotype 6.

21 January 2018

1 reference

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Efficacy of an adeno-associated virus 8-pseudotyped vector in glycogen storage disease type II.

21 January 2018

1 reference

https://api.crossref.org/works/10.1038/SJ.GT.3302550

Efficacy of gene therapy for a prototypical lysosomal storage disease (GSD-II) is critically dependent on vector dose, transgene promoter, and the tissues targeted for vector transduction

21 January 2018

1 reference

https://api.crossref.org/works/10.1038/SJ.GT.3302550

Conditional tissue-specific expression of the acid alpha-glucosidase (GAA) gene in the GAA knockout mice: implications for therapy

21 January 2018

1 reference

https://api.crossref.org/works/10.1038/SJ.GT.3302550

Glycogen stored in skeletal but not in cardiac muscle in acid alpha-glucosidase mutant (Pompe) mice is highly resistant to transgene-encoded human enzyme

21 January 2018

1 reference

https://api.crossref.org/works/10.1038/SJ.GT.3302550

Replacing acid alpha-glucosidase in Pompe disease: recombinant and transgenic enzymes are equipotent, but neither completely clears glycogen from type II muscle fibers.

21 January 2018

1 reference

https://api.crossref.org/works/10.1038/SJ.GT.3302550

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/SJ.GT.3302550

Impact of humoral immune response on distribution and efficacy of recombinant adeno-associated virus-derived acid alpha-glucosidase in a model of glycogen storage disease type II.

21 January 2018

1 reference

https://api.crossref.org/works/10.1038/SJ.GT.3302550

Improved hepatic gene transfer by using an adeno-associated virus serotype 5 vector

21 January 2018

1 reference

https://api.crossref.org/works/10.1038/SJ.GT.3302550

Robust Adenoviral and Adeno-Associated Viral Gene Transfer to the In Vivo Murine Heart

21 January 2018

1 reference

https://api.crossref.org/works/10.1038/SJ.GT.3302550

Infectious Clones and Vectors Derived from Adeno-Associated Virus (AAV) Serotypes Other Than AAV Type 2

21 January 2018

1 reference

https://api.crossref.org/works/10.1038/SJ.GT.3302550

inferred from DOI database lookup

7 January 2021

based on heuristic

Identifiers

DOI

10.1038/SJ.GT.3302550

1 reference

Dimensions Publication ID

14 December 2017

1003581761

0 references

1 reference