Evasion of immune responses to introduced human acid alpha-glucosidase by liver-restricted expression in glycogen storage disease type II. (Q45259698)

11 December 2017

Evasion of immune responses to introduced human acid alpha-glucosidase by liver-restricted expression in glycogen storage disease type II. (English)

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11 December 2017

glycogen storage disease

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Luis M Franco

1

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11 December 2017

Baodong Sun

2

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11 December 2017

Xiaoyi Yang

3

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11 December 2017

Andrew Bird

4

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11 December 2017

Haoyue Zhang

5

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11 December 2017

Ayn Schneider

6

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11 December 2017

Talmage Brown

7

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11 December 2017

Sarah P Young

8

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11 December 2017

Timothy M Clay

9

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11 December 2017

Andrea Amalfitano

10

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11 December 2017

Y T Chen

11

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11 December 2017

Dwight D Koeberl

12

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11 December 2017

6 July 2005

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11 December 2017

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11 December 2017

12

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11 December 2017

5

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11 December 2017

876-884

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Sequence of the cDNA and 5'-flanking region for human acid alpha-glucosidase, detection of an intron in the 5' untranslated leader sequence, definition of 18-bp polymorphisms, and differences with previous cDNA and amino acid sequences

11 December 2017

1 reference

https://api.crossref.org/works/10.1016%2FJ.YMTHE.2005.04.024

Characterization of the human lysosomal alpha-glucosidase gene

7 January 2021

1 reference

https://api.crossref.org/works/10.1016%2FJ.YMTHE.2005.04.024

Murine acid alpha-glucosidase: cell-specific mRNA differential expression during development and maturation

7 January 2021

1 reference

https://api.crossref.org/works/10.1016%2FJ.YMTHE.2005.04.024

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

7 January 2021

1 reference

https://api.crossref.org/works/10.1016%2FJ.YMTHE.2005.04.024

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

7 January 2021

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https://api.crossref.org/works/10.1016%2FJ.YMTHE.2005.04.024

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

7 January 2021

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https://api.crossref.org/works/10.1016%2FJ.YMTHE.2005.04.024

Correction of glycogen storage disease type II by enzyme replacement with a recombinant human acid maltase produced by over-expression in a CHO-DHFR(neg) cell line

7 January 2021

1 reference

https://api.crossref.org/works/10.1016%2FJ.YMTHE.2005.04.024

Enzyme replacement therapy in the mouse model of Pompe disease

7 January 2021

1 reference

https://api.crossref.org/works/10.1016%2FJ.YMTHE.2005.04.024

Immune response to enzyme replacement therapy in lysosomal storage disorder patients and animal models.

7 January 2021

1 reference

https://api.crossref.org/works/10.1016%2FJ.YMTHE.2005.04.024

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

7 January 2021

1 reference

https://api.crossref.org/works/10.1016%2FJ.YMTHE.2005.04.024

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

7 January 2021

1 reference

https://api.crossref.org/works/10.1016%2FJ.YMTHE.2005.04.024

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

7 January 2021

1 reference

https://api.crossref.org/works/10.1016%2FJ.YMTHE.2005.04.024

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

7 January 2021

1 reference

https://api.crossref.org/works/10.1016%2FJ.YMTHE.2005.04.024

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

7 January 2021

1 reference

https://api.crossref.org/works/10.1016%2FJ.YMTHE.2005.04.024

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

7 January 2021

1 reference

https://api.crossref.org/works/10.1016%2FJ.YMTHE.2005.04.024

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

7 January 2021

1 reference

https://api.crossref.org/works/10.1016%2FJ.YMTHE.2005.04.024

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

7 January 2021

1 reference

https://api.crossref.org/works/10.1016%2FJ.YMTHE.2005.04.024

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.

7 January 2021

1 reference

https://api.crossref.org/works/10.1016%2FJ.YMTHE.2005.04.024

Phenotypic correction of a mouse model of hemophilia A using AAV2 vectors encoding the heavy and light chains of FVIII.

7 January 2021

1 reference

https://api.crossref.org/works/10.1016%2FJ.YMTHE.2005.04.024

Total correction of hemophilia A mice with canine FVIII using an AAV 8 serotype.

7 January 2021

1 reference

https://api.crossref.org/works/10.1016%2FJ.YMTHE.2005.04.024

Sustained correction of bleeding disorder in hemophilia B mice by gene therapy

7 January 2021

1 reference

https://api.crossref.org/works/10.1016%2FJ.YMTHE.2005.04.024

AAV2 vector harboring a liver-restricted promoter facilitates sustained expression of therapeutic levels of alpha-galactosidase A and the induction of immune tolerance in Fabry mice.

7 January 2021

1 reference

https://api.crossref.org/works/10.1016%2FJ.YMTHE.2005.04.024

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

7 January 2021

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https://api.crossref.org/works/10.1016%2FJ.YMTHE.2005.04.024

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

7 January 2021

1 reference

https://api.crossref.org/works/10.1016%2FJ.YMTHE.2005.04.024

Quantitation of a urinary tetrasaccharide by gas chromatography and mass spectrometry

7 January 2021

1 reference

https://api.crossref.org/works/10.1016%2FJ.YMTHE.2005.04.024

Liquid chromatographic assay for a glucose tetrasaccharide, a putative biomarker for the diagnosis of Pompe disease

7 January 2021

1 reference

https://api.crossref.org/works/10.1016%2FJ.YMTHE.2005.04.024

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

7 January 2021

1 reference

https://api.crossref.org/works/10.1016%2FJ.YMTHE.2005.04.024

Sustained phenotypic correction of hemophilia B dogs with a factor IX null mutation by liver-directed gene therapy

7 January 2021

1 reference

https://api.crossref.org/works/10.1016%2FJ.YMTHE.2005.04.024

Induction of immune tolerance to coagulation factor IX antigen by in vivo hepatic gene transfer

7 January 2021

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https://api.crossref.org/works/10.1016%2FJ.YMTHE.2005.04.024

Local control of the immune response in the liver

7 January 2021

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https://api.crossref.org/works/10.1016%2FJ.YMTHE.2005.04.024

Adeno-associated virus vector-mediated gene transfer into dystrophin-deficient skeletal muscles evokes enhanced immune response against the transgene product

7 January 2021

1 reference

https://api.crossref.org/works/10.1016%2FJ.YMTHE.2005.04.024

Enhanced expression of transgenes from adeno-associated virus vectors with the woodchuck hepatitis virus posttranscriptional regulatory element: implications for gene therapy

7 January 2021

1 reference

https://api.crossref.org/works/10.1016%2FJ.YMTHE.2005.04.024

Muscle-specific promoters may be necessary for adeno-associated virus-mediated gene transfer in the treatment of muscular dystrophies.

7 January 2021

1 reference

https://api.crossref.org/works/10.1016%2FJ.YMTHE.2005.04.024

Long-term intravenous treatment of Pompe disease with recombinant human alpha-glucosidase from milk

7 January 2021

1 reference

https://api.crossref.org/works/10.1016%2FJ.YMTHE.2005.04.024

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

7 January 2021

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https://api.crossref.org/works/10.1016%2FJ.YMTHE.2005.04.024

Induction of tolerance to a recombinant human enzyme, acid alpha-glucosidase, in enzyme deficient knockout mice

7 January 2021

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https://api.crossref.org/works/10.1016%2FJ.YMTHE.2005.04.024

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

7 January 2021

1 reference

https://api.crossref.org/works/10.1016%2FJ.YMTHE.2005.04.024

Analysis of a glucose tetrasaccharide elevated in Pompe disease by stable isotope dilution–electrospray ionization tandem mass spectrometry

7 January 2021

1 reference

https://api.crossref.org/works/10.1016%2FJ.YMTHE.2005.04.024

Enumerating antigen-specific T-cell responses in peripheral blood: a comparison of peptide MHC Tetramer, ELISpot, and intracellular cytokine analysis.

7 January 2021

1 reference

https://api.crossref.org/works/10.1016%2FJ.YMTHE.2005.04.024

10.1016/J.YMTHE.2005.04.024

7 January 2021

Identifiers

DOI

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11 December 2017

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