(Q36040807)

11 August 2017

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title

Gene therapy of metachromatic leukodystrophy (English)

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11 August 2017

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Ulrich Matzner

1

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11 August 2017

Volkmar Gieselmann

series ordinal

2

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11 August 2017

language of work or name

English

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publication date

1 January 2005

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Expert Opinion on Biological Therapy

11 August 2017

published in

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11 August 2017

volume

5

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11 August 2017

issue

1

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11 August 2017

page(s)

55-65

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Sulfatide is a negative regulator of oligodendrocyte differentiation: development in sulfatide-null mice

11 August 2017

cites work

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https://api.crossref.org/works/10.1517%2F14712598.5.1.55

Phenotype of arylsulfatase A-deficient mice: relationship to human metachromatic leukodystrophy

21 January 2018

1 reference

https://api.crossref.org/works/10.1517%2F14712598.5.1.55

Sulfatide storage in visceral organs of arylsulfatase A-deficient mice

21 January 2018

1 reference

https://api.crossref.org/works/10.1517%2F14712598.5.1.55

Lysosomal sulfatide storage in the brain of arylsulfatase A-deficient mice: cellular alterations and topographic distribution

21 January 2018

1 reference

https://api.crossref.org/works/10.1517%2F14712598.5.1.55

Neuromotor alterations and cerebellar deficits in aged arylsulfatase A-deficient transgenic mice

21 January 2018

1 reference

https://api.crossref.org/works/10.1517%2F14712598.5.1.55

Hyperactivity, neuromotor defects, and impaired learning and memory in a mouse model for metachromatic leukodystrophy.

21 January 2018

1 reference

https://api.crossref.org/works/10.1517%2F14712598.5.1.55

Therapeutic response to intravenous infusions of glucocerebrosidase in a patient with Gaucher disease

21 January 2018

1 reference

https://api.crossref.org/works/10.1517%2F14712598.5.1.55

Alpha-L-iduronidase and enzyme replacement therapy for mucopolysaccharidosis I.

21 January 2018

1 reference

https://api.crossref.org/works/10.1517%2F14712598.5.1.55

Quantitative correlation between the residual activity of beta-hexosaminidase A and arylsulfatase A and the severity of the resulting lysosomal storage disease

21 January 2018

1 reference

https://api.crossref.org/works/10.1517%2F14712598.5.1.55

Bone marrow transplantation corrects the enzyme defect in neurons of the central nervous system in a lysosomal storage disease

21 January 2018

1 reference

https://api.crossref.org/works/10.1517%2F14712598.5.1.55

Turnover of resident microglia in the normal adult mouse brain

21 January 2018

1 reference

https://api.crossref.org/works/10.1517%2F14712598.5.1.55

Bone marrow transplantation for lysosomal diseases.

21 January 2018

1 reference

https://api.crossref.org/works/10.1517%2F14712598.5.1.55

Correction of metachromatic leukodystrophy in the mouse model by transplantation of genetically modified hematopoietic stem cells

21 January 2018

1 reference

https://api.crossref.org/works/10.1517%2F14712598.5.1.55

Restoration of arylsulphatase A activity in human-metachromatic-leucodystrophy fibroblasts via retroviral-vector-mediated gene transfer

21 January 2018

1 reference

https://api.crossref.org/works/10.1517%2F14712598.5.1.55

Transduced fibroblasts and metachromatic leukodystrophy lymphocytes transfer arylsulfatase A to myelinating glia and deficient cells in vitro

21 January 2018

1 reference

https://api.crossref.org/works/10.1517%2F14712598.5.1.55

Transduction of fibroblasts and CD34+ progenitors using a selectable retroviral vector containing cDNAs encoding arylsulfatase A and CD24.

21 January 2018

1 reference

https://api.crossref.org/works/10.1517%2F14712598.5.1.55

Long-term expression and transfer of arylsulfatase A into brain of arylsulfatase A-deficient mice transplanted with bone marrow expressing the arylsulfatase A cDNA from a retroviral vector

21 January 2018

1 reference

https://api.crossref.org/works/10.1517%2F14712598.5.1.55

Bone marrow stem cell-based gene transfer in a mouse model for metachromatic leukodystrophy: effects on visceral and nervous system disease manifestations

21 January 2018

1 reference

https://api.crossref.org/works/10.1517%2F14712598.5.1.55

Molecular mechanisms of translation initiation in eukaryotes

21 January 2018

1 reference

https://api.crossref.org/works/10.1517%2F14712598.5.1.55

Secretion of phosphomannosyl-deficient arylsulphatase A and cathepsin D from isolated human macrophages

21 January 2018

1 reference

https://api.crossref.org/works/10.1517%2F14712598.5.1.55

Mannose 6-phosphate receptor-mediated uptake is defective in acid sphingomyelinase-deficient macrophages: implications for Niemann-Pick disease enzyme replacement therapy

21 January 2018

1 reference

https://api.crossref.org/works/10.1517%2F14712598.5.1.55

Absence of metabolic cross-correction in Tay-Sachs cells: implications for gene therapy

21 January 2018

1 reference

https://api.crossref.org/works/10.1517%2F14712598.5.1.55

Bone marrow stem cell gene therapy of arylsulfatase A-deficient mice, using an arylsulfatase A mutant that is hypersecreted from retrovirally transduced donor-type cells

21 January 2018

1 reference

https://api.crossref.org/works/10.1517%2F14712598.5.1.55

Transgene produces massive overexpression of human beta -glucuronidase in mice, lysosomal storage of enzyme, and strain-dependent tumors

21 January 2018

1 reference

https://api.crossref.org/works/10.1517%2F14712598.5.1.55

Overexpression of human alpha-galactosidase A results in its intracellular aggregation, crystallization in lysosomes, and selective secretion

21 January 2018

1 reference

https://api.crossref.org/works/10.1517%2F14712598.5.1.55

Multiple sulfatase deficiency is caused by mutations in the gene encoding the human C(alpha)-formylglycine generating enzyme

21 January 2018

1 reference

https://api.crossref.org/works/10.1517%2F14712598.5.1.55

Overexpression of N-acetylgalactosamine-4-sulphatase induces a multiple sulphatase deficiency in mucopolysaccharidosis-type-VI fibroblasts

21 January 2018

1 reference

https://api.crossref.org/works/10.1517%2F14712598.5.1.55

21 January 2018

Identifiers

DOI

10.1517/14712598.5.1.55

1 reference

11 August 2017

1 reference