Physiological correction of Pompe disease by systemic delivery of adeno-associated virus serotype 1 vectors (Q45408008)

14 December 2017

title

Physiological correction of Pompe disease by systemic delivery of adeno-associated virus serotype 1 vectors (English)

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6

Melissa A Lewis

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Cathryn Mah

1

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Christina A Pacak

2

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Kerry O Cresawn

3

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Lara R Deruisseau

4

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Sean Germain

5

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Denise A Cloutier

7

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David D Fuller

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Barry J Byrne

9

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publication date

23 January 2007

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volume

15

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issue

3

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page(s)

501-507

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alpha-Glucosidase deficiency in generalized glycogenstorage disease (Pompe's disease).

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cites work

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https://api.crossref.org/works/10.1038%2FSJ.MT.6300100

7 January 2021

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

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https://api.crossref.org/works/10.1038%2FSJ.MT.6300100

7 January 2021

Glycogenosis type II (acid maltase deficiency)

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https://api.crossref.org/works/10.1038%2FSJ.MT.6300100

7 January 2021

Electrocardiographic response to enzyme replacement therapy for Pompe disease.

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https://api.crossref.org/works/10.1038%2FSJ.MT.6300100

7 January 2021

Diagnostic challenges for Pompe disease: an under-recognized cause of floppy baby syndrome

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https://api.crossref.org/works/10.1038%2FSJ.MT.6300100

7 January 2021

Pompe disease diagnosis and management guideline.

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https://api.crossref.org/works/10.1038%2FSJ.MT.6300100

7 January 2021

Safety and efficacy of recombinant acid alpha-glucosidase (rhGAA) in patients with classical infantile Pompe disease: results of a phase II clinical trial

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https://api.crossref.org/works/10.1038%2FSJ.MT.6300100

7 January 2021

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

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https://api.crossref.org/works/10.1038%2FSJ.MT.6300100

7 January 2021

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.

1 reference

https://api.crossref.org/works/10.1038%2FSJ.MT.6300100

7 January 2021

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

1 reference

https://api.crossref.org/works/10.1038%2FSJ.MT.6300100

7 January 2021

Sustained correction of glycogen storage disease type II using adeno-associated virus serotype 1 vectors

1 reference

https://api.crossref.org/works/10.1038%2FSJ.MT.6300100

7 January 2021

Rescue of enzyme deficiency in embryonic diaphragm in a mouse model of metabolic myopathy: Pompe disease

1 reference

https://api.crossref.org/works/10.1038%2FSJ.MT.6300100

7 January 2021

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

1 reference

https://api.crossref.org/works/10.1038%2FSJ.MT.6300100

7 January 2021

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

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7 January 2021

New recombinant serotypes of AAV vectors.

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7 January 2021

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

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7 January 2021

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

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https://api.crossref.org/works/10.1038%2FSJ.MT.6300100

7 January 2021

Body-wide gene therapy of Duchenne muscular dystrophy in the mdx mouse model.

1 reference

https://api.crossref.org/works/10.1038%2FSJ.MT.6300100

7 January 2021

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

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https://api.crossref.org/works/10.1038%2FSJ.MT.6300100

7 January 2021

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

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https://api.crossref.org/works/10.1038%2FSJ.MT.6300100

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The conduction system in Pompe's disease

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https://api.crossref.org/works/10.1038%2FSJ.MT.6300100

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Glycogen stored in skeletal but not in cardiac muscle in acid alpha-glucosidase mutant (Pompe) mice is highly resistant to transgene-encoded human enzyme

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https://api.crossref.org/works/10.1038%2FSJ.MT.6300100

7 January 2021

Current status of cardiac MRI in small animals.

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https://api.crossref.org/works/10.1038%2FSJ.MT.6300100

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Developmental changes of cardiac function and mass assessed with MRI in neonatal, juvenile, and adult mice.

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https://api.crossref.org/works/10.1038%2FSJ.MT.6300100

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Course of disability and respiratory function in untreated late-onset Pompe disease

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https://api.crossref.org/works/10.1038%2FSJ.MT.6300100

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Respiratory failure in Pompe disease: treatment with noninvasive ventilation

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https://api.crossref.org/works/10.1038%2FSJ.MT.6300100

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Pulmonary function assessment by whole-body plethysmography in restrained versus unrestrained mice

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https://api.crossref.org/works/10.1038%2FSJ.MT.6300100

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Respiratory insufficiency and limb muscle weakness in adults with Pompe's disease

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https://api.crossref.org/works/10.1038%2FSJ.MT.6300100

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POMPE'S DISEASE (DIFFUSE GLYCOGENOSIS) WITH NEURONAL STORAGE

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https://api.crossref.org/works/10.1038%2FSJ.MT.6300100

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Production and purification of serotype 1, 2, and 5 recombinant adeno-associated viral vectors

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https://api.crossref.org/works/10.1038%2FSJ.MT.6300100

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Identifiers

DOI

10.1038/SJ.MT.6300100

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14 December 2017

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