(Q45864407)

English

AAV1 mediated co-expression of formylglycine-generating enzyme and arylsulfatase a efficiently corrects sulfatide storage in a mouse model of metachromatic leukodystrophy

scientific article published in January 2007

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scholarly article

1 reference

Europe PubMed Central

17 December 2017

http://europepmc.org/abstract/MED/17164773

AAV1 mediated co-expression of formylglycine-generating enzyme and arylsulfatase a efficiently corrects sulfatide storage in a mouse model of metachromatic leukodystrophy (English)

1 reference

Europe PubMed Central

17 December 2017

http://europepmc.org/abstract/MED/17164773

author name string

Toshiyuki Kurai

1

1 reference

Europe PubMed Central

17 December 2017

http://europepmc.org/abstract/MED/17164773

Sanae Hisayasu

2

1 reference

Europe PubMed Central

17 December 2017

http://europepmc.org/abstract/MED/17164773

Ryo Kitagawa

3

1 reference

Europe PubMed Central

17 December 2017

http://europepmc.org/abstract/MED/17164773

Makoto Migita

4

1 reference

Europe PubMed Central

17 December 2017

http://europepmc.org/abstract/MED/17164773

Hidenori Suzuki

5

1 reference

Europe PubMed Central

17 December 2017

http://europepmc.org/abstract/MED/17164773

Yukihiko Hirai

6

1 reference

Europe PubMed Central

17 December 2017

http://europepmc.org/abstract/MED/17164773

Takashi Shimada

7

1 reference

Europe PubMed Central

17 December 2017

http://europepmc.org/abstract/MED/17164773

language of work or name

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publication date

1 January 2007

1 reference

Europe PubMed Central

17 December 2017

http://europepmc.org/abstract/MED/17164773

Molecular Therapy

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Europe PubMed Central

17 December 2017

http://europepmc.org/abstract/MED/17164773

15

1 reference

Europe PubMed Central

17 December 2017

http://europepmc.org/abstract/MED/17164773

1

1 reference

Europe PubMed Central

17 December 2017

http://europepmc.org/abstract/MED/17164773

38-43

1 reference

Europe PubMed Central

17 December 2017

http://europepmc.org/abstract/MED/17164773

Allogeneic stem cell transplantation for the treatment of lysosomal and peroxisomal metabolic diseases

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Enzyme replacement improves nervous system pathology and function in a mouse model for metachromatic leukodystrophy

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Gene therapy for lysosomal storage diseases.

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Enzyme-replacement therapy for metabolic storage disorders

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The multiple sulfatase deficiency gene encodes an essential and limiting factor for the activity of sulfatases

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

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Conversion of cysteine to formylglycine: a protein modification in the endoplasmic reticulum

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Posttranslational formation of formylglycine in prokaryotic sulfatases by modification of either cysteine or serine

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Characterization of posttranslational formylglycine formation by luminal components of the endoplasmic reticulum

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Coexpression of formylglycine-generating enzyme is essential for synthesis and secretion of functional arylsulfatase A in a mouse model of metachromatic leukodystrophy

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

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

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

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Sulfatide storage in visceral organs of arylsulfatase A-deficient mice

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In vivo gene therapy of metachromatic leukodystrophy by lentiviral vectors: correction of neuropathology and protection against learning impairments in affected mice

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Intracerebral adeno-associated virus-mediated gene transfer in rapidly progressive forms of metachromatic leukodystrophy.

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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

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Axons mediate the distribution of arylsulfatase A within the mouse hippocampus upon gene delivery.

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Distribution of a lysosomal enzyme in the adult brain by axonal transport and by cells of the rostral migratory stream.

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Intrathecal administration of AAV vectors for the treatment of lysosomal storage in the brains of MPS I mice

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Expression vector system based on the chicken beta-actin promoter directs efficient production of interleukin-5.

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Novel adeno-associated viruses from rhesus monkeys as vectors for human gene therapy

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Purification of recombinant adeno-associated virus by iodixanol gradient ultracentrifugation allows rapid and reproducible preparation of vector stocks for gene transfer in the nervous system

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Identifiers

10.1038/SJ.MT.6300012

1 reference

Europe PubMed Central

17 December 2017

http://europepmc.org/abstract/MED/17164773

1 reference

Europe PubMed Central

17 December 2017

http://europepmc.org/abstract/MED/17164773

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