(Q44388027)

English

AAV-mediated expression of galactocerebrosidase in brain results in attenuated symptoms and extended life span in murine models of globoid cell leukodystrophy

scientific article published in May 2005

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scholarly article

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Europe PubMed Central

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2 December 2017

AAV-mediated expression of galactocerebrosidase in brain results in attenuated symptoms and extended life span in murine models of globoid cell leukodystrophy (English)

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Europe PubMed Central

PubMed publication ID

2 December 2017

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based on heuristic

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based on heuristic

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author name string

Mohammad A Rafi

1

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Europe PubMed Central

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2 December 2017

Han Zhi Rao

2

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2 December 2017

Marco A Passini

3

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2 December 2017

Mark Curtis

4

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2 December 2017

Marie T Vanier

5

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2 December 2017

Mariam Zaka

6

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Europe PubMed Central

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2 December 2017

Paola Luzi

7

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Europe PubMed Central

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2 December 2017

John H Wolfe

8

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2 December 2017

David A Wenger

9

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2 December 2017

publication date

1 May 2005

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2 December 2017

Molecular Therapy

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2 December 2017

11

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2 December 2017

5

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2 December 2017

734-744

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Europe PubMed Central

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2 December 2017

Insights into the diagnosis and treatment of lysosomal storage diseases

1 reference

https://api.crossref.org/works/10.1016%2FJ.YMTHE.2004.12.020

7 January 2021

Globoid cell leucodystrophy (Krabbe's disease): deficiency of galactocerebroside beta-galactosidase

1 reference

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Twenty five years of the "psychosine hypothesis": a personal perspective of its history and present status

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Psychosine-induced apoptosis in a mouse oligodendrocyte progenitor cell line is mediated by caspase activation

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Molecular basis of late-life globoid cell leukodystrophy

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7 January 2021

Multiple mutations in the GALC gene in a patient with adult-onset Krabbe disease

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7 January 2021

Molecular heterogeneity of Krabbe disease

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The future for treatment by bone marrow transplantation for adrenoleukodystrophy, metachromatic leukodystrophy, globoid cell leukodystrophy and Hurler syndrome

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Neuropsychological outcomes of several storage diseases with and without bone marrow transplantation

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7 January 2021

Hematopoietic stem-cell transplantation in globoid-cell leukodystrophy

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7 January 2021

Murine, canine and non-human primate models of Krabbe disease

1 reference

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7 January 2021

The twitcher mouse: a model for Krabbe disease and for experimental therapies

1 reference

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7 January 2021

Molecular cloning and expression of cDNA for murine galactocerebrosidase and mutation analysis of the twitcher mouse, a model of Krabbe's disease

1 reference

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7 January 2021

Generation of a mouse with low galactocerebrosidase activity by gene targeting: a new model of globoid cell leukodystrophy (Krabbe disease).

1 reference

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7 January 2021

Prolonged survival and remyelination after hematopoietic cell transplantation in the twitcher mouse.

1 reference

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7 January 2021

Hematopoietic cell transplantation in murine globoid cell leukodystrophy (the twitcher mouse): effects on levels of galactosylceramidase, psychosine, and galactocerebrosides

1 reference

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7 January 2021

Effect of bone marrow transplantation on enzyme levels and clinical course in the neurologically affected twitcher mouse

1 reference

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7 January 2021

Donor-derived cells in the central nervous system of twitcher mice after bone marrow transplantation

1 reference

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7 January 2021

Galactocerebrosidase from human urine: purification and partial characterization

1 reference

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7 January 2021

Purification and characterization of galactocerebrosidase from human lymphocytes

1 reference

https://api.crossref.org/works/10.1016%2FJ.YMTHE.2004.12.020

7 January 2021

Cloning and expression of cDNA encoding human galactocerebrosidase, the enzyme deficient in globoid cell leukodystrophy

1 reference

https://api.crossref.org/works/10.1016%2FJ.YMTHE.2004.12.020

7 January 2021

Krabbe disease: isolation and characterization of a full-length cDNA for human galactocerebrosidase

1 reference

https://api.crossref.org/works/10.1016%2FJ.YMTHE.2004.12.020

7 January 2021

Cloning of the canine GALC cDNA and identification of the mutation causing globoid cell leukodystrophy in West Highland White and Cairn terriers

1 reference

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7 January 2021

Characterization of the rhesus monkey galactocerebrosidase (GALC) cDNA and gene and identification of the mutation causing globoid cell leukodystrophy (Krabbe disease) in this primate

1 reference

https://api.crossref.org/works/10.1016%2FJ.YMTHE.2004.12.020

7 January 2021

Structure and organization of the human galactocerebrosidase (GALC) gene

1 reference

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7 January 2021

Analysis of the 5' flanking region of the human galactocerebrosidase (GALC) gene

1 reference

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7 January 2021

Human galactocerebrosidase gene: promoter analysis of the 5'-flanking region and structural organization

1 reference

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7 January 2021

Retroviral vector-mediated transfer of the galactocerebrosidase (GALC) cDNA leads to overexpression and transfer of GALC activity to neighboring cells

1 reference

https://api.crossref.org/works/10.1016%2FJ.YMTHE.2004.12.020

7 January 2021

Correction of the galactocerebrosidase deficiency in globoid cell leukodystrophy-cultured cells by SL3-3 retroviral-mediated gene transfer

1 reference

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7 January 2021

Transduction of cultured oligodendrocytes from normal and twitcher mice by a retroviral vector containing human galactocerebrosidase (GALC) cDNA.

1 reference

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7 January 2021

Retrovirus-mediated gene transfer and galactocerebrosidase uptake into twitcher glial cells results in appropriate localization and phenotype correction

1 reference

https://api.crossref.org/works/10.1016%2FJ.YMTHE.2004.12.020

7 January 2021

Retroviral-mediated transfer of the galactocerebrosidase gene in neural progenitor cells

1 reference

https://api.crossref.org/works/10.1016%2FJ.YMTHE.2004.12.020

7 January 2021

Intraventricular administration of recombinant adenovirus to neonatal twitcher mouse leads to clinicopathological improvements

1 reference

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7 January 2021

Recombinant AAV viral vectors pseudotyped with viral capsids from serotypes 1, 2, and 5 display differential efficiency and cell tropism after delivery to different regions of the central nervous system

1 reference

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7 January 2021

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

1 reference

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7 January 2021

Intraventricular brain injection of adeno-associated virus type 1 (AAV1) in neonatal mice results in complementary patterns of neuronal transduction to AAV2 and total long-term correction of storage lesions in the brains of beta-glucuronidase-defici

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Differential and persistent expression patterns of CNS gene transfer by an adeno-associated virus (AAV) vector

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

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Adeno-associated virus vector-mediated transduction in the cat brain

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Hybrid vectors based on adeno-associated virus serotypes 2 and 5 for muscle-directed gene transfer

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Widespread gene delivery and structure-specific patterns of expression in the brain after intraventricular injections of neonatal mice with an adeno-associated virus vector

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Neural progenitor cell engraftment corrects lysosomal storage throughout the MPS VII mouse brain

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Analysis of galactocerebrosidase activity in the mouse brain by a new histological staining method.

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10.1016/J.YMTHE.2004.12.020

1 reference

Europe PubMed Central

PubMed publication ID

2 December 2017

PubMed publication ID

1 reference

Europe PubMed Central

PubMed publication ID

2 December 2017

ResearchGate publication ID

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