Bone marrow transplantation increases efficacy of central nervous system-directed enzyme replacement therapy in the murine model of globoid cell leukodystrophy (Q36240279)

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English	Bone marrow transplantation increases efficacy of central nervous system-directed enzyme replacement therapy in the murine model of globoid cell leukodystrophy	scientific article

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5 February 2020

Bone marrow transplantation increases efficacy of central nervous system-directed enzyme replacement therapy in the murine model of globoid cell leukodystrophy (English)

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5 February 2020

enzyme replacement therapy

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7

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5 February 2020

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Elizabeth Y Qin

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5 February 2020

Jacqueline A Hawkins-Salsbury

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Xuntian Jiang

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Adarsh S Reddy

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Nuri B Farber

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Daniel S Ory

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1 June 2012

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Molecular Genetics and Metabolism

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107

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186-196

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1-2

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11 June 2022

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Bone Marrow Transplantation Alters the Tremor Phenotype in the Murine Model of Globoid-Cell Leukodystrophy.

1 reference

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1 October 2017

Bone marrow transplantation augments the effect of brain- and spinal cord-directed adeno-associated virus 2/5 gene therapy by altering inflammation in the murine model of globoid-cell leukodystrophy

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=3444533

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Combination therapies for lysosomal storage disease: is the whole greater than the sum of its parts?

1 reference

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1 October 2017

Single-dose intracerebroventricular administration of galactocerebrosidase improves survival in a mouse model of globoid cell leukodystrophy

1 reference

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1 October 2017

Enzyme replacement therapy results in substantial improvements in early clinical phenotype in a mouse model of globoid cell leukodystrophy

1 reference

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Transplantation of umbilical-cord blood in babies with infantile Krabbe's disease

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Molecular mechanism of psychosine-induced cell death in human oligodendrocyte cell line

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

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'Green mice' as a source of ubiquitous green cells.

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

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Distribution and characterization of GFP(+) donor hematogenous cells in Twitcher mice after bone marrow transplantation

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Kinetics of central nervous system microglial and macrophage engraftment: analysis using a transgenic bone marrow transplantation model.

1 reference

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

1 reference

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Promising CNS-directed enzyme replacement therapy for lysosomal storage diseases

1 reference

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Intracerebroventricular infusion of acid sphingomyelinase corrects CNS manifestations in a mouse model of Niemann-Pick A disease

1 reference

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Central nervous system-directed AAV2/5-mediated gene therapy synergizes with bone marrow transplantation in the murine model of globoid-cell leukodystrophy

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

1 reference

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Early use of drastic therapy

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

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

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Substrate-reduction therapy enhances the benefits of bone marrow transplantation in young mice with globoid cell leukodystrophy

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An apoptotic depletion of oligodendrocytes in the twitcher, a murine model of globoid cell leukodystrophy.

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Treatment of murine mucopolysaccharidosis type VII by syngeneic bone marrow transplantation in neonates.

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

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22 September 2018

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

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Hereditary leucodystrophy in the mouse: the new mutant twitcher

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The Twitcher mouse: an enzymatically authentic model of human globoid cell leukodystrophy (Krabbe disease)

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Hematopoietic stem-cell transplantation in globoid-cell leukodystrophy

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Inborn errors of mucopolysaccharide metabolism

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Infiltration of hematogenous lineage cells into the demyelinating central nervous system of twitcher mice

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Percutaneous intravenous injection in neonatal mice

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10.1016/J.YMGME.2012.05.021

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5 February 2020

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5 February 2020

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