(Q45008585)

8 December 2017

title

Efficacy of helper-dependent adenovirus vector-mediated gene therapy in murine glycogen storage disease type Ia. (English)

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8 December 2017

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glycogen storage disease

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Dwight D Koeberl

1

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8 December 2017

B Sun

2

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8 December 2017

A Bird

3

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8 December 2017

Y T Chen

4

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8 December 2017

K Oka

5

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8 December 2017

L Chan

6

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8 December 2017

publication date

15 May 2007

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8 December 2017

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8 December 2017

volume

15

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8 December 2017

issue

7

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8 December 2017

page(s)

1253-1258

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Pulmonary hypertension in glycogen storage disease type I

8 December 2017

cites work

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https://api.crossref.org/works/10.1038%2FSJ.MT.6300188

7 January 2021

Glycogen storage disease in adults

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7 January 2021

Progressive pulmonary hypertension: a fatal complication of type I glycogen storage disease

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7 January 2021

Hepatocellular carcinoma in glycogen storage disease type Ia: a case series

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7 January 2021

Glucose-6-phosphatase dependent substrate transport in the glycogen storage disease type-1a mouse

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7 January 2021

Correction of glycogen storage disease type 1a in a mouse model by gene therapy

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https://api.crossref.org/works/10.1038%2FSJ.MT.6300188

7 January 2021

Sustained hepatic and renal glucose-6-phosphatase expression corrects glycogen storage disease type Ia in mice

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https://api.crossref.org/works/10.1038%2FSJ.MT.6300188

7 January 2021

Long-term correction of murine glycogen storage disease type Ia by recombinant adeno-associated virus-1-mediated gene transfer

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https://api.crossref.org/works/10.1038%2FSJ.MT.6300188

7 January 2021

Early, sustained efficacy of adeno-associated virus vector-mediated gene therapy in glycogen storage disease type Ia.

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https://api.crossref.org/works/10.1038%2FSJ.MT.6300188

7 January 2021

In vivo hepatic gene therapy: complete albeit transient correction of factor IX deficiency in hemophilia B dogs

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https://api.crossref.org/works/10.1038%2FSJ.MT.6300188

7 January 2021

Immune responses to transgene-encoded proteins limit the stability of gene expression after injection of replication-defective adenovirus vectors

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https://api.crossref.org/works/10.1038%2FSJ.MT.6300188

7 January 2021

Lifetime correction of genetic deficiency in mice with a single injection of helper-dependent adenoviral vector

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https://api.crossref.org/works/10.1038%2FSJ.MT.6300188

7 January 2021

An adenoviral vector deleted for all viral coding sequences results in enhanced safety and extended expression of a leptin transgene

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https://api.crossref.org/works/10.1038%2FSJ.MT.6300188

7 January 2021

Use of helper-dependent adenoviral vectors of alternative serotypes permits repeat vector administration

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https://api.crossref.org/works/10.1038%2FSJ.MT.6300188

7 January 2021

Glycogen storage disease type Ia in two littermate Maltese puppies.

1 reference

https://api.crossref.org/works/10.1038%2FSJ.MT.6300188

7 January 2021

A new mutation of the glucose-6-phosphatase gene in a 4-year-old girl with oligosymptomatic glycogen storage disease type 1a

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7 January 2021

Therapeutic factor VIII levels and negligible toxicity in mouse and dog models of hemophilia A following gene therapy with high-capacity adenoviral vectors

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https://api.crossref.org/works/10.1038%2FSJ.MT.6300188

7 January 2021

A new adenoviral helper-dependent vector results in long-term therapeutic levels of human coagulation factor IX at low doses in vivo

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https://api.crossref.org/works/10.1038%2FSJ.MT.6300188

7 January 2021

Long-Term Stable Expression of Human Apolipoprotein A-I Mediated by Helper-Dependent Adenovirus Gene Transfer Inhibits Atherosclerosis Progression and Remodels Atherosclerotic Plaques in a Mouse Model of Familial Hypercholesterolemia

1 reference

https://api.crossref.org/works/10.1038%2FSJ.MT.6300188

7 January 2021

Lifelong elimination of hyperbilirubinemia in the Gunn rat with a single injection of helper-dependent adenoviral vector

1 reference

https://api.crossref.org/works/10.1038%2FSJ.MT.6300188

7 January 2021

Improved hepatic transduction, reduced systemic vector dissemination, and long-term transgene expression by delivering helper-dependent adenoviral vectors into the surgically isolated liver of nonhuman primates.

1 reference

https://api.crossref.org/works/10.1038%2FSJ.MT.6300188

7 January 2021

Developing adenoviral-mediated in vivo gene therapy for ornithine transcarbamylase deficiency

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7 January 2021

Fatal systemic inflammatory response syndrome in a ornithine transcarbamylase deficient patient following adenoviral gene transfer

1 reference

https://api.crossref.org/works/10.1038%2FSJ.MT.6300188

7 January 2021

Acute toxicity after high-dose systemic injection of helper-dependent adenoviral vectors into nonhuman primates.

1 reference

https://api.crossref.org/works/10.1038%2FSJ.MT.6300188

7 January 2021

Long-term stable correction of low-density lipoprotein receptor-deficient mice with a helper-dependent adenoviral vector expressing the very low-density lipoprotein receptor

1 reference

https://api.crossref.org/works/10.1038%2FSJ.MT.6300188

7 January 2021

Woodchuck hepatitis virus contains a tripartite posttranscriptional regulatory element.

1 reference

https://api.crossref.org/works/10.1038%2FSJ.MT.6300188

7 January 2021

Canine model and genomic structural organization of glycogen storage disease type Ia (GSD Ia).

1 reference

https://api.crossref.org/works/10.1038%2FSJ.MT.6300188

7 January 2021

Optimization of the helper-dependent adenovirus system for production and potency in vivo

1 reference

https://api.crossref.org/works/10.1038%2FSJ.MT.6300188

7 January 2021

Low-density lipoprotein receptor gene therapy using helper-dependent adenovirus produces long-term protection against atherosclerosis in a mouse model of familial hypercholesterolemia

1 reference

https://api.crossref.org/works/10.1038%2FSJ.MT.6300188

7 January 2021

A helper-dependent adenovirus vector system: removal of helper virus by Cre-mediated excision of the viral packaging signal

1 reference

https://api.crossref.org/works/10.1038%2FSJ.MT.6300188

7 January 2021

Delivery of glucose-6-phosphatase in a canine model for glycogen storage disease, type Ia, with adeno-associated virus (AAV) vectors

1 reference

https://api.crossref.org/works/10.1038%2FSJ.MT.6300188

7 January 2021

Identifiers

DOI

10.1038/SJ.MT.6300188

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8 December 2017

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