Long-term enzyme correction and lipid reduction in multiple organs of primary and secondary transplanted Fabry mice receiving transduced bone marrow cells
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Local and global cerebral blood flow and glucose utilization in the alpha-galactosidase A knockout mouse model of Fabry diseaseGene therapy: prospects for glycolipid storage diseases.Human mesenchymal stem cells in rodent whole-embryo culture are reprogrammed to contribute to kidney tissuesLentivector Iterations and Pre-Clinical Scale-Up/Toxicity Testing: Targeting Mobilized CD34+ Cells for Correction of Fabry DiseaseUse of lissamine rhodamine ceramide trihexoside as a functional assay for alpha-galactosidase A in intact cellsA symptomatic Fabry disease mouse model generated by inducing globotriaosylceramide synthesis.Long-term systemic therapy of Fabry disease in a knockout mouse by adeno-associated virus-mediated muscle-directed gene transfer.Adeno-associated viral vector-mediated gene transfer results in long-term enzymatic and functional correction in multiple organs of Fabry mice.Increased globotriaosylceramide levels in a transgenic mouse expressing human alpha1,4-galactosyltransferase and a mouse model for treating Fabry disease.Migalastat HCl reduces globotriaosylsphingosine (lyso-Gb3) in Fabry transgenic mice and in the plasma of Fabry patients.α-Galactosidase A expressed in the salivary glands partially corrects organ biochemical deficits in the fabry mouse through endocrine traffickingPreselective gene therapy for Fabry disease.Genetically altered mouse models: the good, the bad, and the ugly.Sex differences of urinary and kidney globotriaosylceramide and lyso-globotriaosylceramide in Fabry mice.Gene therapy for neurological disease.Cellular and tissue distribution of intravenously administered agalsidase alfa.Distributions of Globotriaosylceramide Isoforms, and Globotriaosylsphingosine and Its Analogues in an α-Galactosidase A Knockout Mouse, a Model of Fabry Disease.Lentivector transduction improves outcomes over transplantation of human HSCs alone in NOD/SCID/Fabry mice.Fundamental research is the basis for understanding and treatment of many human diseases.Novel therapeutic targets for the treatment of Fabry disease.Bioluminescent imaging of a marking transgene and correction of Fabry mice by neonatal injection of recombinant lentiviral vectors.Gene therapy for fabry disease: a review of the literature.Fabry disease: experience of screening dialysis patients for Fabry disease.Alpha-galactosidase A-Tat fusion enhances storage reduction in hearts and kidneys of Fabry mice.Optimization of Culture Conditions for Some Identified Fungal Species and Stability Profile of α-Galactosidase Produced.Sequencing and characterization of the porcine α-galactosidase A gene: towards the generation of a porcine model for Fabry disease.Efficient correction of Fabry mice and patient cells mediated by lentiviral transduction of hematopoietic stem/progenitor cells.Minimum requirement of donor cells to reduce the glycolipid storage following bone marrow transplantation in a murine model of Fabry disease.Efficient transfer of PSA and PSMA cDNAs into DCs generates antibody and T cell antitumor responses in vivo.Fabry disease
P2860
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P2860
Long-term enzyme correction and lipid reduction in multiple organs of primary and secondary transplanted Fabry mice receiving transduced bone marrow cells
description
2000 թուականի Յունիսին հրատարակուած գիտական յօդուած
@hyw
2000 թվականի հունիսին հրատարակված գիտական հոդված
@hy
article publié dans les Procee ...... f the United States of America
@fr
artículu científicu espublizáu en 2000
@ast
im Juni 2000 veröffentlichter wissenschaftlicher Artikel
@de
scientific journal article
@en
vedecký článok (publikovaný 2000/06/20)
@sk
vědecký článek publikovaný v roce 2000
@cs
wetenschappelijk artikel (gepubliceerd op 2000/06/20)
@nl
наукова стаття, опублікована в червні 2000
@uk
name
Long-term enzyme correction an ...... g transduced bone marrow cells
@ast
Long-term enzyme correction an ...... g transduced bone marrow cells
@en
Long-term enzyme correction an ...... g transduced bone marrow cells
@nl
type
label
Long-term enzyme correction an ...... g transduced bone marrow cells
@ast
Long-term enzyme correction an ...... g transduced bone marrow cells
@en
Long-term enzyme correction an ...... g transduced bone marrow cells
@nl
prefLabel
Long-term enzyme correction an ...... g transduced bone marrow cells
@ast
Long-term enzyme correction an ...... g transduced bone marrow cells
@en
Long-term enzyme correction an ...... g transduced bone marrow cells
@nl
P2093
P2860
P356
P1476
Long-term enzyme correction an ...... g transduced bone marrow cells
@en
P2093
A. B. Kulkarni
G. J. Murray
J. A. Medin
J. M. Quirk
R. O. Brady
T. Ohshima
T. Takenaka
P2860
P304
P356
10.1073/PNAS.120177997
P407
P577
2000-06-20T00:00:00Z