Correction of glycogen storage disease type II by an adeno-associated virus vector containing a muscle-specific promoter (Q45457148)

14 December 2017

Correction of glycogen storage disease type II by an adeno-associated virus vector containing a muscle-specific promoter (English)

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14 December 2017

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glycogen storage disease

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Baodong Sun

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14 December 2017

Haoyue Zhang

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14 December 2017

Luis M Franco

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14 December 2017

Talmage Brown

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14 December 2017

Andrew Bird

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14 December 2017

Ayn Schneider

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14 December 2017

Dwight D Koeberl

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14 December 2017

1 June 2005

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14 December 2017

889-898

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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

14 December 2017

1 reference

https://api.crossref.org/works/10.1016%2FJ.YMTHE.2005.01.012

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.2005.01.012

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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https://api.crossref.org/works/10.1016%2FJ.YMTHE.2005.01.012

Enzyme replacement therapy in the mouse model of Pompe disease

7 January 2021

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https://api.crossref.org/works/10.1016%2FJ.YMTHE.2005.01.012

Complete correction of acid alpha-glucosidase deficiency in Pompe disease fibroblasts in vitro, and lysosomally targeted expression in neonatal rat cardiac and skeletal muscle

7 January 2021

1 reference

https://api.crossref.org/works/10.1016%2FJ.YMTHE.2005.01.012

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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https://api.crossref.org/works/10.1016%2FJ.YMTHE.2005.01.012

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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https://api.crossref.org/works/10.1016%2FJ.YMTHE.2005.01.012

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.2005.01.012

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

1 reference

https://api.crossref.org/works/10.1016%2FJ.YMTHE.2005.01.012

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.2005.01.012

Systemic delivery of genes to striated muscles using adeno-associated viral vectors.

7 January 2021

1 reference

https://api.crossref.org/works/10.1016%2FJ.YMTHE.2005.01.012

Immune responses to dystropin: implications for gene therapy of Duchenne muscular dystrophy

7 January 2021

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https://api.crossref.org/works/10.1016%2FJ.YMTHE.2005.01.012

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.2005.01.012

Analysis of muscle creatine kinase regulatory elements in recombinant adenoviral vectors

7 January 2021

1 reference

https://api.crossref.org/works/10.1016%2FJ.YMTHE.2005.01.012

E-box sites and a proximal regulatory region of the muscle creatine kinase gene differentially regulate expression in diverse skeletal muscles and cardiac muscle of transgenic mice

7 January 2021

1 reference

https://api.crossref.org/works/10.1016%2FJ.YMTHE.2005.01.012

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.2005.01.012

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.2005.01.012

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

7 January 2021

1 reference

https://api.crossref.org/works/10.1016%2FJ.YMTHE.2005.01.012

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.2005.01.012

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.2005.01.012

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.2005.01.012

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

7 January 2021

1 reference

https://api.crossref.org/works/10.1016%2FJ.YMTHE.2005.01.012

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

7 January 2021

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https://api.crossref.org/works/10.1016%2FJ.YMTHE.2005.01.012

Risk and prevention of anti-factor IX formation in AAV-mediated gene transfer in the context of a large deletion of F9.

7 January 2021

1 reference

https://api.crossref.org/works/10.1016%2FJ.YMTHE.2005.01.012

Muscle-directed gene transfer and transient immune suppression result in sustained partial correction of canine hemophilia B caused by a null mutation

7 January 2021

1 reference

https://api.crossref.org/works/10.1016%2FJ.YMTHE.2005.01.012

Influence of vector dose on factor IX-specific T and B cell responses in muscle-directed gene therapy

7 January 2021

1 reference

https://api.crossref.org/works/10.1016%2FJ.YMTHE.2005.01.012

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

7 January 2021

1 reference

https://api.crossref.org/works/10.1016%2FJ.YMTHE.2005.01.012

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

1 reference

https://api.crossref.org/works/10.1016%2FJ.YMTHE.2005.01.012

Scaleable chromatographic purification process for recombinant adeno-associated virus (rAAV).

7 January 2021

1 reference

https://api.crossref.org/works/10.1016%2FJ.YMTHE.2005.01.012

Scalable purification of adeno-associated virus type 2, 4, or 5 using ion-exchange chromatography

7 January 2021

1 reference

https://api.crossref.org/works/10.1016%2FJ.YMTHE.2005.01.012

Purification of recombinant adeno-associated virus vectors by column chromatography and its performance in vivo

7 January 2021

1 reference

https://api.crossref.org/works/10.1016%2FJ.YMTHE.2005.01.012

Optimized production of high-titer recombinant adeno-associated virus in roller bottles

7 January 2021

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https://api.crossref.org/works/10.1016%2FJ.YMTHE.2005.01.012

High-titer recombinant adeno-associated virus production from replicating amplicons and herpes vectors deleted for glycoprotein H.

7 January 2021

1 reference

https://api.crossref.org/works/10.1016%2FJ.YMTHE.2005.01.012

Insect cells as a factory to produce adeno-associated virus type 2 vectors

7 January 2021

1 reference

https://api.crossref.org/works/10.1016%2FJ.YMTHE.2005.01.012

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

https://api.crossref.org/works/10.1016%2FJ.YMTHE.2005.01.012

A simplified system for generating recombinant adenoviruses

7 January 2021

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https://api.crossref.org/works/10.1016%2FJ.YMTHE.2005.01.012

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.2005.01.012

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

7 January 2021

1 reference

https://api.crossref.org/works/10.1016%2FJ.YMTHE.2005.01.012

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

7 January 2021

1 reference

https://api.crossref.org/works/10.1016%2FJ.YMTHE.2005.01.012

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

7 January 2021

1 reference

https://api.crossref.org/works/10.1016%2FJ.YMTHE.2005.01.012

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

7 January 2021

1 reference

https://api.crossref.org/works/10.1016%2FJ.YMTHE.2005.01.012

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.2005.01.012

Infectious clones and vectors derived from adeno-associated virus (AAV) serotypes other than AAV type 2.

7 January 2021

1 reference

https://api.crossref.org/works/10.1016%2FJ.YMTHE.2005.01.012

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7 January 2021

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DOI

10.1016/J.YMTHE.2005.01.012

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14 December 2017

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