Modulation of glycogen synthesis by RNA interference: towards a new therapeutic approach for glycogenosis type II. (Q39941399)

15 September 2017

Modulation of glycogen synthesis by RNA interference: towards a new therapeutic approach for glycogenosis type II. (English)

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15 September 2017

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Gaelle Douillard-Guilloux

1

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15 September 2017

Nina Raben

2

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15 September 2017

Shoichi Takikita

3

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15 September 2017

Lionel Batista

4

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15 September 2017

Catherine Caillaud

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15 September 2017

Emmanuel Richard

6

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15 September 2017

9 September 2008

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15 September 2017

Human Molecular Genetics

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15 September 2017

17

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15 September 2017

24

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15 September 2017

3876-3886

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Pompe disease diagnosis and management guideline.

15 September 2017

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https://api.crossref.org/works/10.1093%2FHMG%2FDDN290

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

21 January 2018

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https://api.crossref.org/works/10.1093%2FHMG%2FDDN290

Enzyme therapy for pompe disease with recombinant human alpha-glucosidase from rabbit milk

21 January 2018

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https://api.crossref.org/works/10.1093%2FHMG%2FDDN290

Glycogen storage disease types I and II: treatment updates

21 January 2018

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https://api.crossref.org/works/10.1093%2FHMG%2FDDN290

Enzyme therapy for Pompe disease: from science to industrial enterprise

21 January 2018

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https://api.crossref.org/works/10.1093%2FHMG%2FDDN290

Enzyme replacement therapy in the mouse model of Pompe disease

21 January 2018

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https://api.crossref.org/works/10.1093%2FHMG%2FDDN290

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

21 January 2018

1 reference

https://api.crossref.org/works/10.1093%2FHMG%2FDDN290

Replacing acid alpha-glucosidase in Pompe disease: recombinant and transgenic enzymes are equipotent, but neither completely clears glycogen from type II muscle fibers.

21 January 2018

1 reference

https://api.crossref.org/works/10.1093%2FHMG%2FDDN290

Both type 1 and type 2a muscle fibers can respond to enzyme therapy in Pompe disease.

21 January 2018

1 reference

https://api.crossref.org/works/10.1093%2FHMG%2FDDN290

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

21 January 2018

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https://api.crossref.org/works/10.1093%2FHMG%2FDDN290

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

21 January 2018

1 reference

https://api.crossref.org/works/10.1093%2FHMG%2FDDN290

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

21 January 2018

1 reference

https://api.crossref.org/works/10.1093%2FHMG%2FDDN290

Improved efficacy of gene therapy approaches for Pompe disease using a new, immune-deficient GSD-II mouse model

21 January 2018

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https://api.crossref.org/works/10.1093%2FHMG%2FDDN290

Oral maintenance clinical trial with miglustat for type I Gaucher disease: switch from or combination with intravenous enzyme replacement.

21 January 2018

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https://api.crossref.org/works/10.1093%2FHMG%2FDDN290

The role of the iminosugar N-butyldeoxynojirimycin (miglustat) in the management of type I (non-neuronopathic) Gaucher disease: a position statement

21 January 2018

1 reference

https://api.crossref.org/works/10.1093%2FHMG%2FDDN290

Miglustat for treatment of Niemann-Pick C disease: a randomised controlled study

21 January 2018

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https://api.crossref.org/works/10.1093%2FHMG%2FDDN290

Regulation of glycogen synthesis in human muscle cells.

21 January 2018

1 reference

https://api.crossref.org/works/10.1093%2FHMG%2FDDN290

Glycogenin: the primer for mammalian and yeast glycogen synthesis

21 January 2018

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https://api.crossref.org/works/10.1093%2FHMG%2FDDN290

In vivo regulation of muscle glycogen synthase and the control of glycogen synthesis

21 January 2018

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https://api.crossref.org/works/10.1093%2FHMG%2FDDN290

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

21 January 2018

1 reference

https://api.crossref.org/works/10.1093%2FHMG%2FDDN290

Atrophy of S6K1(-/-) skeletal muscle cells reveals distinct mTOR effectors for cell cycle and size control.

21 January 2018

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https://api.crossref.org/works/10.1093%2FHMG%2FDDN290

Increases in glycogenin and glycogenin mRNA accompany glycogen resynthesis in human skeletal muscle

21 January 2018

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https://api.crossref.org/works/10.1093%2FHMG%2FDDN290

De novo synthesis of bacterial glycogen: Agrobacterium tumefaciens glycogen synthase is involved in glucan initiation and elongation

21 January 2018

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https://api.crossref.org/works/10.1093%2FHMG%2FDDN290

Autophagy and lysosomes in Pompe disease

21 January 2018

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https://api.crossref.org/works/10.1093%2FHMG%2FDDN290

Immune tolerance induction to enzyme-replacement therapy by co-administration of short-term, low-dose methotrexate in a murine Pompe disease model

21 January 2018

1 reference

https://api.crossref.org/works/10.1093%2FHMG%2FDDN290

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

21 January 2018

1 reference

https://api.crossref.org/works/10.1093%2FHMG%2FDDN290

Abnormal cardiac development in the absence of heart glycogen

21 January 2018

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https://api.crossref.org/works/10.1093%2FHMG%2FDDN290

Exercise capacity of mice genetically lacking muscle glycogen synthase: in mice, muscle glycogen is not essential for exercise

21 January 2018

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https://api.crossref.org/works/10.1093%2FHMG%2FDDN290

Glucose metabolism in mice lacking muscle glycogen synthase

21 January 2018

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https://api.crossref.org/works/10.1093%2FHMG%2FDDN290

New insights into impaired muscle glycogen synthesis

21 January 2018

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https://api.crossref.org/works/10.1093%2FHMG%2FDDN290

Cardiomyopathy and exercise intolerance in muscle glycogen storage disease 0

21 January 2018

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https://api.crossref.org/works/10.1093%2FHMG%2FDDN290

Enhanced survival in Sandhoff disease mice receiving a combination of substrate deprivation therapy and bone marrow transplantation.

21 January 2018

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https://api.crossref.org/works/10.1093%2FHMG%2FDDN290

Temporal neuropathologic and behavioral phenotype of 6neo/6neo Pompe disease mice

21 January 2018

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https://api.crossref.org/works/10.1093%2FHMG%2FDDN290

21 January 2018

Identifiers

DOI

10.1093/HMG/DDN290

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15 September 2017

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