Long-term correction of glycogen storage disease type II with a hybrid Ad-AAV vector (Q40669141)

22 September 2017

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

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22 September 2017

Y-T Chen

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22 September 2017

Andrew Bird

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22 September 2017

Andrea Amalfitano

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22 September 2017

Dwight D Koeberl

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22 September 2017

1 February 2003

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22 September 2017

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7

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22 September 2017

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

1 reference

https://api.crossref.org/works/10.1016%2FS1525-0016%2802%2900055-2

7 January 2021

Generalized glycogen storage and cardiomegaly in a knockout mouse model of Pompe disease

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https://api.crossref.org/works/10.1016%2FS1525-0016%2802%2900055-2

7 January 2021

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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https://api.crossref.org/works/10.1016%2FS1525-0016%2802%2900055-2

7 January 2021

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

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https://api.crossref.org/works/10.1016%2FS1525-0016%2802%2900055-2

7 January 2021

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

1 reference

https://api.crossref.org/works/10.1016%2FS1525-0016%2802%2900055-2

7 January 2021

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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https://api.crossref.org/works/10.1016%2FS1525-0016%2802%2900055-2

7 January 2021

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

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https://api.crossref.org/works/10.1016%2FS1525-0016%2802%2900055-2

7 January 2021

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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https://api.crossref.org/works/10.1016%2FS1525-0016%2802%2900055-2

7 January 2021

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

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https://api.crossref.org/works/10.1016%2FS1525-0016%2802%2900055-2

7 January 2021

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.

1 reference

https://api.crossref.org/works/10.1016%2FS1525-0016%2802%2900055-2

7 January 2021

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

1 reference

https://api.crossref.org/works/10.1016%2FS1525-0016%2802%2900055-2

7 January 2021

Stable gene transfer and expression of human blood coagulation factor IX after intramuscular injection of recombinant adeno-associated virus

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https://api.crossref.org/works/10.1016%2FS1525-0016%2802%2900055-2

7 January 2021

Persistent and therapeutic concentrations of human factor IX in mice after hepatic gene transfer of recombinant AAV vectors

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https://api.crossref.org/works/10.1016%2FS1525-0016%2802%2900055-2

7 January 2021

Persistent, therapeutically relevant levels of human granulocyte colony-stimulating factor in mice after systemic delivery of adeno-associated virus vectors.

1 reference

https://api.crossref.org/works/10.1016%2FS1525-0016%2802%2900055-2

7 January 2021

Correction of hemophilia B in canine and murine models using recombinant adeno-associated viral vectors

1 reference

https://api.crossref.org/works/10.1016%2FS1525-0016%2802%2900055-2

7 January 2021

Long-term correction of canine hemophilia B by gene transfer of blood coagulation factor IX mediated by adeno-associated viral vector

1 reference

https://api.crossref.org/works/10.1016%2FS1525-0016%2802%2900055-2

7 January 2021

Evaluation of the transfer and expression in mice of an enzyme-encoding gene using a human adenovirus vector

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https://api.crossref.org/works/10.1016%2FS1525-0016%2802%2900055-2

7 January 2021

Long term correction of bilirubin-UDP-glucuronosyltransferase deficiency in Gunn rats by administration of a recombinant adenovirus during the neonatal period

1 reference

https://api.crossref.org/works/10.1016%2FS1525-0016%2802%2900055-2

7 January 2021

Successful expression of human factor IX following repeat administration of adenoviral vector in mice.

1 reference

https://api.crossref.org/works/10.1016%2FS1525-0016%2802%2900055-2

7 January 2021

Adenovirus mediated expression of therapeutic plasma levels of human factor IX in mice

1 reference

https://api.crossref.org/works/10.1016%2FS1525-0016%2802%2900055-2

7 January 2021

Implication of interfering antibody formation and apoptosis as two different mechanisms leading to variable duration of adenovirus-mediated transgene expression in immune-competent mice

1 reference

https://api.crossref.org/works/10.1016%2FS1525-0016%2802%2900055-2

7 January 2021

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

1 reference

https://api.crossref.org/works/10.1016%2FS1525-0016%2802%2900055-2

7 January 2021

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

1 reference

https://api.crossref.org/works/10.1016%2FS1525-0016%2802%2900055-2

7 January 2021

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

1 reference

https://api.crossref.org/works/10.1016%2FS1525-0016%2802%2900055-2

7 January 2021

Forced myofiber regeneration promotes dystrophin gene transfer and improved muscle function despite advanced disease in old dystrophic mice

1 reference

https://api.crossref.org/works/10.1016%2FS1525-0016%2802%2900055-2

7 January 2021

Isolation and characterization of packaging cell lines that coexpress the adenovirus E1, DNA polymerase, and preterminal proteins: implications for gene therapy

1 reference

https://api.crossref.org/works/10.1016%2FS1525-0016%2802%2900055-2

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

1 reference

https://api.crossref.org/works/10.1016%2FS1525-0016%2802%2900055-2

7 January 2021

Introns increase transcriptional efficiency in transgenic mice

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https://api.crossref.org/works/10.1016%2FS1525-0016%2802%2900055-2

7 January 2021

A simplified system for generating recombinant adenoviruses

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https://api.crossref.org/works/10.1016%2FS1525-0016%2802%2900055-2

7 January 2021

Multiply deleted [E1, polymerase-, and pTP-] adenovirus vector persists despite deletion of the preterminal protein

1 reference

https://api.crossref.org/works/10.1016%2FS1525-0016%2802%2900055-2

7 January 2021

Production and characterization of improved adenovirus vectors with the E1, E2b, and E3 genes deleted

1 reference

https://api.crossref.org/works/10.1016%2FS1525-0016%2802%2900055-2

7 January 2021

Quantification of murine cytokine mRNAs using real time quantitative reverse transcriptase PCR

1 reference

https://api.crossref.org/works/10.1016%2FS1525-0016%2802%2900055-2

7 January 2021

10.1016/S1525-0016(02)00055-2

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DOI

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22 September 2017

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