Reversal of pathology in murine mucopolysaccharidosis type VII by somatic cell gene transfer
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Correction of mucopolysaccharidosis type IIIb fibroblasts by lentiviral vector-mediated gene transferGene therapy for mucopolysaccharidosisalpha-Galactosidase A deficient mice: a model of Fabry diseaseTherapeutic neonatal hepatic gene therapy in mucopolysaccharidosis VII dogs.Systemic and central nervous system correction of lysosomal storage in mucopolysaccharidosis type VII miceIntracisternal A-particle element transposition into the murine beta-glucuronidase gene correlates with loss of enzyme activity: a new model for beta-glucuronidase deficiency in the C3H mouseTherapeutic efficacy of bone marrow transplant, intracranial AAV-mediated gene therapy, or both in the mouse model of MPS IIIBEnzyme replacement therapy for murine mucopolysaccharidosis type VIIBehavioral consequences of bone marrow transplantation in the treatment of murine mucopolysaccharidosis type VII.Dysregulation of gene expression in a lysosomal storage disease varies between brain regions implicating unexpected mechanisms of neuropathology.Correction of lysosomal storage in the liver and spleen of MPS VII mice by implantation of genetically modified skin fibroblasts.Active site mutant transgene confers tolerance to human beta-glucuronidase without affecting the phenotype of MPS VII mice.Adeno-associated viral vector-mediated gene transfer results in long-term enzymatic and functional correction in multiple organs of Fabry mice.Widespread nonhematopoietic tissue distribution by transplanted human progenitor cells with high aldehyde dehydrogenase activity.Combination therapies for lysosomal storage disease: is the whole greater than the sum of its parts?Production of MPS VII mouse (Gus(tm(hE540A x mE536A)Sly)) doubly tolerant to human and mouse beta-glucuronidaseLysosomal storage disease: gene therapy on both sides of the blood-brain barrier.Hematopoietic stem cell gene therapy: progress toward therapeutic targets.Gene therapy for neurologic manifestations of mucopolysaccharidoses.Gene therapy approaches to prolonging corneal allograft survival.Adenovirus-mediated gene transfer and expression of human beta-glucuronidase gene in the liver, spleen, and central nervous system in mucopolysaccharidosis type VII mice.A concise peer into the background, initial thoughts and practices of human gene therapy.Partial rescue of mucopolysaccharidosis type VII mice with a lifelong engraftment of allogeneic stem cells in uteroA single-base-pair deletion in the beta-glucuronidase gene accounts for the phenotype of murine mucopolysaccharidosis type VIIMetabolic correction and cross-correction of mucopolysaccharidosis type II (Hunter syndrome) by retroviral-mediated gene transfer and expression of human iduronate-2-sulfataseNeonatal gene transfer leads to widespread correction of pathology in a murine model of lysosomal storage disease.Mesenchymal stem cells for the sustained in vivo delivery of bioactive factors.The level of mRNA encoding the amphotropic retrovirus receptor in mouse and human hematopoietic stem cells is low and correlates with the efficiency of retrovirus transduction.Animal models of human disease for gene therapyMurine mucopolysaccharidosis type VII: long term therapeutic effects of enzyme replacement and enzyme replacement followed by bone marrow transplantation.Enzyme replacement therapy for murine mucopolysaccharidosis type VII leads to improvements in behavior and auditory function.Targeted disruption of the arylsulfatase B gene results in mice resembling the phenotype of mucopolysaccharidosis VICorrection in trans for Fabry disease: expression, secretion and uptake of alpha-galactosidase A in patient-derived cells driven by a high-titer recombinant retroviral vector.Long-term in vitro correction of alpha-L-iduronidase deficiency (Hurler syndrome) in human bone marrow.Clinical Improvement of Alpha-mannosidosis Cat Following a Single Cisterna Magna Infusion of AAV1.Lysosomal enzyme replacement therapies: Historical development, clinical outcomes, and future perspectives.Decreased lysosomal storage in the adult MPS VII mouse brain in the vicinity of grafts of retroviral vector-corrected fibroblasts secreting high levels of beta-glucuronidase.Lysosomal storage diseases of animals: an essay in comparative pathology.Lentiviral-transduced human mesenchymal stem cells persistently express therapeutic levels of enzyme in a xenotransplantation model of human diseasePrevention of systemic clinical disease in MPS VII mice following AAV-mediated neonatal gene transfer.
P2860
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P2860
Reversal of pathology in murine mucopolysaccharidosis type VII by somatic cell gene transfer
description
2016 թուականի Դեկտեմբերին հրատարակուած գիտական յօդուած
@hyw
2016 թվականի դեկտեմբերին հրատարակված գիտական հոդված
@hy
article publié dans la revue scientifique Nature
@fr
artículu científicu espublizáu en 2016
@ast
scientific journal article
@en
vedecký článok (publikovaný 2016/12/24)
@sk
vědecký článek publikovaný v roce 2016
@cs
wetenschappelijk artikel (gepubliceerd op 2016/12/24)
@nl
наукова стаття, опублікована в грудні 2016
@uk
مقالة علمية (نشرت في 24-12-2016)
@ar
name
Reversal of pathology in murin ...... by somatic cell gene transfer
@ast
Reversal of pathology in murin ...... by somatic cell gene transfer
@en
Reversal of pathology in murin ...... by somatic cell gene transfer
@nl
type
label
Reversal of pathology in murin ...... by somatic cell gene transfer
@ast
Reversal of pathology in murin ...... by somatic cell gene transfer
@en
Reversal of pathology in murin ...... by somatic cell gene transfer
@nl
prefLabel
Reversal of pathology in murin ...... by somatic cell gene transfer
@ast
Reversal of pathology in murin ...... by somatic cell gene transfer
@en
Reversal of pathology in murin ...... by somatic cell gene transfer
@nl
P2093
P356
P1433
P1476
Reversal of pathology in murin ...... by somatic cell gene transfer
@en
P2093
C. A. Vogler
E. H. Birkenmeier
J. E. Barker
J. H. Wolfe
L. B. Rowe
M. S. Sands
P2888
P304
P356
10.1038/360749A0
P407
P577
2016-12-24T00:00:00Z
P6179
1020719652