Fully deleted adenovirus persistently expressing GAA accomplishes long-term skeletal muscle glycogen correction in tolerant and nontolerant GSD-II mice (Q64378389)

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scientific article published on 05 October 2005

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English	Fully deleted adenovirus persistently expressing GAA accomplishes long-term skeletal muscle glycogen correction in tolerant and nontolerant GSD-II mice	scientific article published on 05 October 2005

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7 January 2020

Fully deleted adenovirus persistently expressing GAA accomplishes long-term skeletal muscle glycogen correction in tolerant and nontolerant GSD-II mice (English)

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7 January 2020

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Anne Kiang

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7 January 2020

Zachary C Hartman

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7 January 2020

Shaoxi Liao

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7 January 2020

Fang Xu

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7 January 2020

Delila Serra

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Donna J Palmer

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7 January 2020

Philip Ng

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7 January 2020

Andrea Amalfitano

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7 January 2020

Molecular Therapy

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127-134

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7 January 2020

Carrier frequency for glycogen storage disease type II in New York and estimates of affected individuals born with the disease

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Towards a molecular therapy for glycogen storage disease type II (Pompe disease).

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A diagnostic protocol for adult-onset glycogen storage disease type II

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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

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7 January 2021

Multiple muscles in the AMD quail can be “cross‐corrected” of pathologic glycogen accumulation after intravenous injection of an [E1‐, polymerase‐] adenovirus vector encoding human acid‐α‐glucosidase

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Persistence of an [E1-, polymerase-] adenovirus vector despite transduction of a neoantigen into immune-competent mice

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7 January 2021

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

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7 January 2021

Primary adenovirus-specific cytotoxic T lymphocyte response occurs after viral clearance and liver enzyme elevation

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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

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7 January 2021

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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7 January 2021

Glycogen storage in multiple muscles of old GSD-II mice can be rapidly cleared after a single intravenous injection with a modified adenoviral vector expressing hGAA.

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7 January 2021

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

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

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

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7 January 2021

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

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7 January 2021

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

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7 January 2021

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

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7 January 2021

Long-term erythropoietin expression in rodents and non-human primates following intramuscular injection of a replication-defective adenoviral vector

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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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7 January 2021

Use of a liver-specific promoter reduces immune response to the transgene in adenoviral vectors

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Intravenous administration of an E1/E3-deleted adenoviral vector induces tolerance to factor IX in C57BL/6 mice

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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.

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Immune evasion by muscle-specific gene expression in dystrophic muscle

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Separating fact from fiction: assessing the potential of modified adenovirus vectors for use in human gene therapy

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Improved system for helper-dependent adenoviral vector production

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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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7 January 2021

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

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Liver toxicities typically induced by first-generation adenoviral vectors can be reduced by use of E1, E2b-deleted adenoviral vectors

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Role of viral antigens in destructive cellular immune responses to adenovirus vector-transduced cells in mouse lungs.

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7 January 2021

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10.1016/J.YMTHE.2005.08.006

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7 January 2020

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Europe PubMed Central

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7 January 2020

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