(Q44624370)

4 December 2017

Enzyme replacement therapy in the mouse model of Pompe disease (English)

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enzyme replacement therapy

4 December 2017

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author

Abigail L. Gilbert

3

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4 December 2017

6

Thurberg BL

1 reference

4 December 2017

7

Mattaliano RJ

1 reference

4 December 2017

Raben N

1

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4 December 2017

Danon M

2

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4 December 2017

Dwivedi S

4

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4 December 2017

Collins B

5

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4 December 2017

Nagaraju K

8

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4 December 2017

Plotz PH

9

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4 December 2017

1 September 2003

1 reference

Molecular Genetics and Metabolism

4 December 2017

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4 December 2017

80

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4 December 2017

1-2

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4 December 2017

159-169

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Replacement therapy for inherited enzyme deficiency--macrophage-targeted glucocerebrosidase for Gaucher's disease

4 December 2017

1 reference

https://api.crossref.org/works/10.1016%2FJ.YMGME.2003.08.022

Enzyme replacement and enhancement therapies: lessons from lysosomal disorders

7 January 2021

1 reference

https://api.crossref.org/works/10.1016%2FJ.YMGME.2003.08.022

Enzyme replacement therapy for the mucopolysaccharide storage disorders

7 January 2021

1 reference

https://api.crossref.org/works/10.1016%2FJ.YMGME.2003.08.022

Enzyme therapy for Pompe disease: from science to industrial enterprise

7 January 2021

1 reference

https://api.crossref.org/works/10.1016%2FJ.YMGME.2003.08.022

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

7 January 2021

1 reference

https://api.crossref.org/works/10.1016%2FJ.YMGME.2003.08.022

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

7 January 2021

1 reference

https://api.crossref.org/works/10.1016%2FJ.YMGME.2003.08.022

Identification of two subtypes of infantile acid maltase deficiency

7 January 2021

1 reference

https://api.crossref.org/works/10.1016%2FJ.YMGME.2003.08.022

Enzyme therapy in type 1 Gaucher disease: comparative efficacy of mannose-terminated glucocerebrosidase from natural and recombinant sources

7 January 2021

1 reference

https://api.crossref.org/works/10.1016%2FJ.YMGME.2003.08.022

Lysosomal storage diseases

7 January 2021

1 reference

https://api.crossref.org/works/10.1016%2FJ.YMGME.2003.08.022

Structure and function of the mannose 6-phosphate/insulinlike growth factor II receptors

7 January 2021

1 reference

https://api.crossref.org/works/10.1016%2FJ.YMGME.2003.08.022

Expression and routeing of human lysosomal alpha-glucosidase in transiently transfected mammalian cells

7 January 2021

1 reference

https://api.crossref.org/works/10.1016%2FJ.YMGME.2003.08.022

High-level production of recombinant human lysosomal acid alpha-glucosidase in Chinese hamster ovary cells which targets to heart muscle and corrects glycogen accumulation in fibroblasts from patients with Pompe disease

7 January 2021

1 reference

https://api.crossref.org/works/10.1016%2FJ.YMGME.2003.08.022

Recombinant human acid alpha-glucosidase: high level production in mouse milk, biochemical characteristics, correction of enzyme deficiency in GSDII KO mice

7 January 2021

1 reference

https://api.crossref.org/works/10.1016%2FJ.YMGME.2003.08.022

Recombinant human alpha-glucosidase from rabbit milk in Pompe patients.

7 January 2021

1 reference

https://api.crossref.org/works/10.1016%2FJ.YMGME.2003.08.022

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

7 January 2021

1 reference

https://api.crossref.org/works/10.1016%2FJ.YMGME.2003.08.022

Recombinant human acid alpha-glucosidase enzyme therapy for infantile glycogen storage disease type II: results of a phase I/II clinical trial

7 January 2021

1 reference

https://api.crossref.org/works/10.1016%2FJ.YMGME.2003.08.022

Morphological changes in muscle tissue of patients with infantile Pompe's disease receiving enzyme replacement therapy

7 January 2021

1 reference

https://api.crossref.org/works/10.1016%2FJ.YMGME.2003.08.022

Clinical and metabolic correction of pompe disease by enzyme therapy in acid maltase-deficient quail

7 January 2021

1 reference

https://api.crossref.org/works/10.1016%2FJ.YMGME.2003.08.022

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

7 January 2021

1 reference

https://api.crossref.org/works/10.1016%2FJ.YMGME.2003.08.022

Molecular cloning of acid alpha-glucosidase cDNA of Japanese quail (Coturnix coturnix japonica) and the lack of its mRNA in acid maltase deficient quails

7 January 2021

1 reference

https://api.crossref.org/works/10.1016%2FJ.YMGME.2003.08.022

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

7 January 2021

1 reference

https://api.crossref.org/works/10.1016%2FJ.YMGME.2003.08.022

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

7 January 2021

1 reference

https://api.crossref.org/works/10.1016%2FJ.YMGME.2003.08.022

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

7 January 2021

1 reference

https://api.crossref.org/works/10.1016%2FJ.YMGME.2003.08.022

Surprises of genetic engineering: a possible model of polyglucosan body disease

7 January 2021

1 reference

https://api.crossref.org/works/10.1016%2FJ.YMGME.2003.08.022

Mannose 6-phosphate receptors in sorting and transport of lysosomal enzymes

7 January 2021

1 reference

https://api.crossref.org/works/10.1016%2FJ.YMGME.2003.08.022

Systemic correction of the muscle disorder glycogen storage disease type II after hepatic targeting of a modified adenovirus vector encoding human acid-alpha-glucosidase

7 January 2021

1 reference

https://api.crossref.org/works/10.1016%2FJ.YMGME.2003.08.022

Correction of glycogen storage disease type II by enzyme replacement with a recombinant human acid maltase produced by over-expression in a CHO-DHFR(neg) cell line

7 January 2021

1 reference

https://api.crossref.org/works/10.1016%2FJ.YMGME.2003.08.022

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

7 January 2021

1 reference

https://api.crossref.org/works/10.1016%2FJ.YMGME.2003.08.022

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

7 January 2021

1 reference

https://api.crossref.org/works/10.1016%2FJ.YMGME.2003.08.022

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

7 January 2021

1 reference

https://api.crossref.org/works/10.1016%2FJ.YMGME.2003.08.022

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

7 January 2021

1 reference

https://api.crossref.org/works/10.1016%2FJ.YMGME.2003.08.022

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

7 January 2021

1 reference

https://api.crossref.org/works/10.1016%2FJ.YMGME.2003.08.022

Long-term correction of glycogen storage disease type II with a hybrid Ad-AAV vector

7 January 2021

1 reference

https://api.crossref.org/works/10.1016%2FJ.YMGME.2003.08.022

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

7 January 2021

1 reference

https://api.crossref.org/works/10.1016%2FJ.YMGME.2003.08.022

Protein transport from the secretory to the endocytic pathway in mammalian cells

7 January 2021

1 reference

https://api.crossref.org/works/10.1016%2FJ.YMGME.2003.08.022

Human acid alpha-glucosidase from rabbit milk has therapeutic effect in mice with glycogen storage disease type II

7 January 2021

1 reference

https://api.crossref.org/works/10.1016%2FJ.YMGME.2003.08.022

Long-term and high-dose trials of enzyme replacement therapy in the canine model of mucopolysaccharidosis I.

7 January 2021

1 reference

https://api.crossref.org/works/10.1016%2FJ.YMGME.2003.08.022

Fabry disease: preclinical studies demonstrate the effectiveness of alpha-galactosidase A replacement in enzyme-deficient mice

7 January 2021

1 reference

https://api.crossref.org/works/10.1016%2FJ.YMGME.2003.08.022

The influence of lysosomes on glycogen metabolism

7 January 2021

1 reference

https://api.crossref.org/works/10.1016%2FJ.YMGME.2003.08.022

10.1016/J.YMGME.2003.08.022

7 January 2021

Identifiers

DOI

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4 December 2017

1 reference