Efficacy of an adeno-associated virus 8-pseudotyped vector in glycogen storage disease type II. (Q40483404)

20 September 2017

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

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20 September 2017

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glycogen storage disease

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Baodong Sun

1

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20 September 2017

Haoyue Zhang

2

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20 September 2017

Luis M Franco

3

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20 September 2017

Sarah P Young

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20 September 2017

Ayn Schneider

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20 September 2017

Andrew Bird

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20 September 2017

Andrea Amalfitano

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20 September 2017

Y-T Chen

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20 September 2017

Dwight D Koeberl

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20 September 2017

1 January 2005

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11

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1

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20 September 2017

57-65

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

20 September 2017

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Adeno-associated virus type 6 (AAV6) vectors mediate efficient transduction of airway epithelial cells in mouse lungs compared to that of AAV2 vectors

7 January 2021

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Cross-packaging of a single adeno-associated virus (AAV) type 2 vector genome into multiple AAV serotypes enables transduction with broad specificity

7 January 2021

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Several log increase in therapeutic transgene delivery by distinct adeno-associated viral serotype vectors

7 January 2021

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

7 January 2021

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Enzyme replacement therapy in the mouse model of Pompe disease

7 January 2021

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

7 January 2021

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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

7 January 2021

1 reference

https://api.crossref.org/works/10.1016%2FJ.YMTHE.2004.10.004

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

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https://api.crossref.org/works/10.1016%2FJ.YMTHE.2004.10.004

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

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

7 January 2021

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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

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

7 January 2021

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

7 January 2021

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https://api.crossref.org/works/10.1016%2FJ.YMTHE.2004.10.004

Quantitation of a urinary tetrasaccharide by gas chromatography and mass spectrometry

7 January 2021

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

7 January 2021

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https://api.crossref.org/works/10.1016%2FJ.YMTHE.2004.10.004

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

7 January 2021

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https://api.crossref.org/works/10.1016%2FJ.YMTHE.2004.10.004

Rapid uncoating of vector genomes is the key to efficient liver transduction with pseudotyped adeno-associated virus vectors

7 January 2021

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Sex significantly influences transduction of murine liver by recombinant adeno-associated viral vectors through an androgen-dependent pathway

7 January 2021

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https://api.crossref.org/works/10.1016%2FJ.YMTHE.2004.10.004

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.2004.10.004

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

7 January 2021

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https://api.crossref.org/works/10.1016%2FJ.YMTHE.2004.10.004

Enzyme replacement and enhancement therapies for lysosomal diseases

7 January 2021

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Clinical investigation in duchenne dystrophy: 2. Determination of the ?power? of therapeutic trials based on the natural history

7 January 2021

1 reference

https://api.crossref.org/works/10.1016%2FJ.YMTHE.2004.10.004

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.2004.10.004

Adeno-associated virus vector carrying human minidystrophin genes effectively ameliorates muscular dystrophy in mdx mouse model

7 January 2021

1 reference

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

7 January 2021

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Direct intramuscular injection with recombinant AAV vectors results in sustained expression in a dog model of hemophilia

7 January 2021

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

7 January 2021

1 reference

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Safety and efficacy of factor IX gene transfer to skeletal muscle in murine and canine hemophilia B models by adeno-associated viral vector serotype 1.

7 January 2021

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

7 January 2021

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

7 January 2021

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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

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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.

7 January 2021

1 reference

https://api.crossref.org/works/10.1016%2FJ.YMTHE.2004.10.004

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

7 January 2021

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

7 January 2021

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

7 January 2021

1 reference

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Improved adeno-associated virus vector production with transfection of a single helper adenovirus gene, E4orf6.

7 January 2021

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Recombinant adeno-associated virus purification using novel methods improves infectious titer and yield

7 January 2021

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Persistent expression of human clotting factor IX from mouse liver after intravenous injection of adeno-associated virus vectors

7 January 2021

1 reference

https://api.crossref.org/works/10.1016%2FJ.YMTHE.2004.10.004

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.2004.10.004

The murine DNA-PKcs gene consists of 86 exons dispersed in more than 250 kb.

7 January 2021

1 reference

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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

7 January 2021

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

7 January 2021

1 reference

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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

7 January 2021

1 reference

https://api.crossref.org/works/10.1016%2FJ.YMTHE.2004.10.004

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

7 January 2021

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https://api.crossref.org/works/10.1016%2FJ.YMTHE.2004.10.004

Transduction by adeno-associated virus vectors in the rabbit airway: efficiency, persistence, and readministration.

7 January 2021

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inferred from DOI database lookup

7 January 2021

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DOI

10.1016/J.YMTHE.2004.10.004

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release_ozzb6rqjsbhidirkdot3wonuum

20 September 2017

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https://api.fatcat.wiki/v0/release/ozzb6rqjsbhidirkdot3wonuum

mapped directly with Wikidata item

24 November 2022

based on heuristic

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