Improved efficacy of gene therapy approaches for Pompe disease using a new, immune-deficient GSD-II mouse model (Q45877711)

17 December 2017

title

Improved efficacy of gene therapy approaches for Pompe disease using a new, immune-deficient GSD-II mouse model (English)

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17 December 2017

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Xu F

1

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17 December 2017

Ding E

2

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17 December 2017

Liao SX

3

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Migone F

4

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17 December 2017

Dai J

5

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17 December 2017

Schneider A

6

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17 December 2017

Serra D

7

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17 December 2017

Chen YT

8

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17 December 2017

Amalfitano A

9

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17 December 2017

publication date

1 November 2004

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17 December 2017

published in

Gene Therapy

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17 December 2017

volume

11

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17 December 2017

issue

21

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17 December 2017

page(s)

1590-1598

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17 December 2017

https://scigraph.springernature.com/pub.10.1038/sj.gt.3302314

exact match

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cites work

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

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Characterization of the human lysosomal alpha-glucosidase gene

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Glycogenosis type II (acid maltase deficiency)

21 January 2018

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21 January 2018

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7 January 2021

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Towards a molecular therapy for glycogen storage disease type II (Pompe disease).

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Recombinant human alpha-glucosidase from rabbit milk in Pompe patients.

21 January 2018

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High-level production of recombinant human lysosomal acid alpha-glucosidase in Chinese hamster ovary cells which targets to heart muscle and corrects glycogen accumulation in fibroblasts from patients with Pompe disease

21 January 2018

1 reference

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Systemic correction of the muscle disorder glycogen storage disease type II after hepatic targeting of a modified adenovirus vector encoding human acid-alpha-glucosidase

21 January 2018

1 reference

https://api.crossref.org/works/10.1038%2FSJ.GT.3302314

Long-term efficacy after [E1-, polymerase-] adenovirus-mediated transfer of human acid-alpha-glucosidase gene into glycogen storage disease type II knockout mice

21 January 2018

1 reference

https://api.crossref.org/works/10.1038%2FSJ.GT.3302314

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%2FSJ.GT.3302314

Intercellular transfer of the virally derived precursor form of acid alpha-glucosidase corrects the enzyme deficiency in inherited cardioskeletal myopathy Pompe disease

21 January 2018

1 reference

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

21 January 2018

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Multiple muscles in the AMD quail can be “cross‐corrected” of pathologic glycogen accumulation after intravenous injection of an [E1‐, polymerase‐] adenovirus vector encoding human acid‐α‐glucosidase

21 January 2018

1 reference

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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

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Identification of a nonsense mutation in the carboxyl-terminal region of DNA-dependent protein kinase catalytic subunit in the scid mouse

21 January 2018

1 reference

https://api.crossref.org/works/10.1038%2FSJ.GT.3302314

Nonsense mutation at Tyr-4046 in the DNA-dependent protein kinase catalytic subunit of severe combined immune deficiency mice

21 January 2018

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Optimization of the helper-dependent adenovirus system for production and potency in vivo

21 January 2018

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Clinical and metabolic correction of pompe disease by enzyme therapy in acid maltase-deficient quail

21 January 2018

1 reference

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

21 January 2018

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Muscle as a putative producer of acid alpha-glucosidase for glycogenosis type II gene therapy

21 January 2018

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Long-term correction of glycogen storage disease type II with a hybrid Ad-AAV vector

21 January 2018

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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

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Lysosomal dysfunction in muscle with special reference to glycogen storage disease type II.

21 January 2018

1 reference

https://api.crossref.org/works/10.1038%2FSJ.GT.3302314

Intravenous administration of phosphorylated acid alpha-glucosidase leads to uptake of enzyme in heart and skeletal muscle of mice

21 January 2018

1 reference

https://api.crossref.org/works/10.1038%2FSJ.GT.3302314

Human acid alpha-glucosidase from rabbit milk has therapeutic effect in mice with glycogen storage disease type II

21 January 2018

1 reference

https://api.crossref.org/works/10.1038%2FSJ.GT.3302314

inferred from DOI database lookup

7 January 2021

based on heuristic

Receptor-mediated uptake of acid alpha-glucosidase corrects lysosomal glycogen storage in cultured skeletal muscle

1 reference

https://api.crossref.org/works/10.1038%2FSJ.GT.3302314

inferred from DOI database lookup

7 January 2021

based on heuristic

Identifiers

DOI

10.1038/SJ.GT.3302314

1 reference

17 December 2017

Dimensions Publication ID

0 references

1 reference

17 December 2017