Correction of glycogenosis type 2 by muscle-specific lentiviral vector (Q39936904)

15 September 2017

title

Correction of glycogenosis type 2 by muscle-specific lentiviral vector (English)

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15 September 2017

Emmanuel Richard

1

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15 September 2017

Gaëlle Douillard-Guilloux

series ordinal

2

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15 September 2017

Lionel Batista

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3

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15 September 2017

Catherine Caillaud

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4

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15 September 2017

publication date

23 September 2008

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In Vitro Cellular & Developmental Biology - Animal

15 September 2017

published in

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15 September 2017

volume

44

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15 September 2017

issue

10

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15 September 2017

page(s)

397-406

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

15 September 2017

cites work

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12 December 2020

Generalized glycogen storage and cardiomegaly in a knockout mouse model of Pompe disease

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12 December 2020

Structure of adeno-associated virus vector DNA following transduction of the skeletal muscle

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12 December 2020

Ability of adeno-associated virus serotype 8-mediated hepatic expression of acid alpha-glucosidase to correct the biochemical and motor function deficits of presymptomatic and symptomatic Pompe mice

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Enhanced response to enzyme replacement therapy in Pompe disease after the induction of immune tolerance

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Gene therapy for severe combined immunodeficiency: are we there yet?

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Enzyme replacement therapy in late-onset Pompe's disease: a three-year follow-up

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12 December 2020

Recombinant human acid [alpha]-glucosidase: major clinical benefits in infantile-onset Pompe disease

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12 December 2020

Breakdown of lysosomal glycogen in cultured fibroblasts from glycogenosis type II patients after uptake of acid alpha-glucosidase

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12 December 2020

Improved expression in hematopoietic and lymphoid cells in mice after transplantation of bone marrow transduced with a modified retroviral vector

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12 December 2020

Woodchuck hepatitis virus posttranscriptional regulatory element enhances expression of transgenes delivered by retroviral vectors.

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Hematopoietic stem cell gene therapy of murine protoporphyria by methylguanine-DNA-methyltransferase-mediated in vivo drug selection

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12 December 2020

The genetic engineering of hematopoietic stem cells: the rise of lentiviral vectors, the conundrum of the ltr, and the promise of lineage-restricted vectors

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12 December 2020

Long-term expression and secretion of human glucocerebrosidase by primary murine and human myoblasts and differentiated myotubes

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12 December 2020

Isolation and Partial Characterization of the Structural Gene for Human Acid Alpha Glucosidase

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12 December 2020

Enhanced efficacy of an AAV vector encoding chimeric, highly secreted acid alpha-glucosidase in glycogen storage disease type II.

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12 December 2020

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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12 December 2020

Genetics of type II glycogenosis: Assignment of the human gene for acid α-glucosidase to chromosome 17

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12 December 2020

Self-inactivating lentivirus vector for safe and efficient in vivo gene delivery

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12 December 2020

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

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12 December 2020

Structural and functional changes of lysosomal acid alpha-glucosidase during intracellular transport and maturation

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12 December 2020

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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12 December 2020

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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12 December 2020

Sustained expression of genes delivered directly into liver and muscle by lentiviral vectors

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12 December 2020

Synthetic muscle promoters: activities exceeding naturally occurring regulatory sequences

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12 December 2020

Evasion of immune responses to introduced human acid alpha-glucosidase by liver-restricted expression in glycogen storage disease type II.

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Autologous transplantation of muscle precursor cells modified with a lentivirus for muscular dystrophy: human cells and primate models

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How not to be seen: immune-evasion strategies in gene therapy.

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12 December 2020

Therapeutic levels of factor IX expression using a muscle-specific promoter and adeno-associated virus serotype 1 vector

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High-level erythroid-specific gene expression in primary human and murine hematopoietic cells with self-inactivating lentiviral vectors

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12 December 2020

Pompe disease: a review of the current diagnosis and treatment recommendations in the era of enzyme replacement therapy

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Retroviral transfer of acid alpha-glucosidase cDNA to enzyme-deficient myoblasts results in phenotypic spread of the genotypic correction by both secretion and fusion

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High-efficiency growth hormone-releasing hormone plasmid vector administration into skeletal muscle mediated by electroporation in pigs

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12 December 2020

Muscle as a putative producer of acid alpha-glucosidase for glycogenosis type II gene therapy

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12 December 2020

Genetic fate of recombinant adeno-associated virus vector genomes in muscle

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12 December 2020

Correlation of acid alpha-glucosidase and glycogen content in skin fibroblasts with age of onset in Pompe disease

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12 December 2020

Enzyme replacement therapy in the mouse model of Pompe disease

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12 December 2020

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

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12 December 2020

10.1007/S11626-008-9138-5

Identifiers

DOI

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15 September 2017

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