(Q40669141)

22 September 2017

title

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

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22 September 2017

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glycogen storage disease

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Bao-dong Sun

1

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22 September 2017

Y-T Chen

2

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22 September 2017

Andrew Bird

3

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22 September 2017

Andrea Amalfitano

4

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22 September 2017

Dwight D Koeberl

5

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22 September 2017

publication date

1 February 2003

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22 September 2017

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22 September 2017

volume

7

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22 September 2017

issue

2

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22 September 2017

page(s)

193-201

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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

22 September 2017

cites work

1 reference

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Recombinant human acid alpha-glucosidase: high level production in mouse milk, biochemical characteristics, correction of enzyme deficiency in GSDII KO mice

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

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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

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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

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

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Adenovirus-mediated transfer of human acid maltase gene reduces glycogen accumulation in skeletal muscle of Japanese quail with acid maltase deficiency

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Neonatal gene transfer leads to widespread correction of pathology in a murine model of lysosomal storage disease.

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Neonatal intramuscular injection with recombinant adeno-associated virus results in prolonged beta-glucuronidase expression in situ and correction of liver pathology in mucopolysaccharidosis type VII mice.

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Muscle-specific overexpression of the adenovirus primary receptor CAR overcomes low efficiency of gene transfer to mature skeletal muscle

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7 January 2021

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7 January 2021

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7 January 2021

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

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7 January 2021

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7 January 2021

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7 January 2021

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Identifiers

DOI

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22 September 2017

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