(Q45884747)

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AAV2/5 vector expressing galactocerebrosidase ameliorates CNS disease in the murine model of globoid-cell leukodystrophy more efficiently than AAV2.

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https://www.ebi.ac.uk/europepmc/webservices/rest/search?query=EXT_ID:15996520%20AND%20SRC:MED&resulttype=core&format=json

13 January 2020

AAV2/5 vector expressing galactocerebrosidase ameliorates CNS disease in the murine model of globoid-cell leukodystrophy more efficiently than AAV2 (English)

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13 January 2020

vector-borne disease

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based on heuristic

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13 January 2020

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Darshong Lin

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13 January 2020

Corinne R Fantz

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13 January 2020

Beth Levy

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13 January 2020

Mohammad A Rafi

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13 January 2020

Carole Vogler

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13 January 2020

David A Wenger

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13 January 2020

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1 September 2005

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13 January 2020

Molecular Therapy

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13 January 2020

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13 January 2020

3

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13 January 2020

422-430

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13 January 2020

Lysosomal storage disorders: diagnostic dilemmas and prospects for therapy

1 reference

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7 January 2021

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

1 reference

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7 January 2021

Molecular genetics of Krabbe disease (globoid cell leukodystrophy): Diagnostic and clinical implications

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7 January 2021

Progressive accumulation of toxic metabolite in a genetic leukodystrophy

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

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

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

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7 January 2021

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

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7 January 2021

Hematopoietic stem-cell transplantation in globoid-cell leukodystrophy

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Twitcher mice with only a single active galactosylceramide synthase gene exhibit clearly detectable but therapeutically minor phenotypic improvements

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7 January 2021

L-cycloserine slows the clinical and pathological course in mice with globoid cell leukodystrophy (twitcher mice)

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7 January 2021

Delayed clinical and pathological signs in twitcher (globoid cell leukodystrophy) mice on a C57BL/6 x CAST/Ei background

1 reference

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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

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

1 reference

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7 January 2021

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

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

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7 January 2021

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

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7 January 2021

Recombinant AAV-mediated gene delivery to the central nervous system.

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7 January 2021

Elimination of lysosomal storage in brains of MPS VII mice treated by intrathecal administration of an adeno-associated virus vector

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7 January 2021

Intracranial injection of recombinant adeno-associated virus improves cognitive function in a murine model of mucopolysaccharidosis type VII.

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7 January 2021

Neurological correction of lysosomal storage in a mucopolysaccharidosis IIIB mouse model by adeno-associated virus-mediated gene delivery

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7 January 2021

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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7 January 2021

Recombinant adeno-associated virus type 2, 4, and 5 vectors: transduction of variant cell types and regions in the mammalian central nervous system.

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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

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

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Transduction of the choroid plexus and ependyma in neonatal mouse brain by vesicular stomatitis virus glycoprotein-pseudotyped lentivirus and adeno-associated virus type 5 vectors

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

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The HIV Tat protein transduction domain improves the biodistribution of beta-glucuronidase expressed from recombinant viral vectors.

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Spacio-temporal progression of demyelination in twitcher mouse: with clinico-pathological correlation

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Proliferation of microglia/macrophages in the demyelinating CNS and PNS of twitcher mouse.

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Immunohistological study of globoid cell leukodystrophy.

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Expression of immune-related molecules is downregulated in twitcher mice following bone marrow transplantation

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Activated microglia in cortex of mouse models of mucopolysaccharidoses I and IIIB

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