Effective cell and gene therapy in a murine model of Gaucher disease. - Wikidata - awgldk - TriplyDB

Effective cell and gene therapy in a murine model of Gaucher disease.

Effective cell and gene therapy in a murine model of Gaucher disease.

http://www.wikidata.org/entity/Q33256447

about

Large animal models of rare genetic disorders: sheep as phenotypically relevant models of human genetic disease Genome-wide association study of N370S homozygous Gaucher disease reveals the candidacy of CLN8 gene as a genetic modifier contributing to extreme phenotypic variation A Novel Functional Missense Mutation p.T219A in Type 1 Gaucher's Disease.Murine models of acute neuronopathic Gaucher disease.Correction of murine ADAMTS13 deficiency by hematopoietic progenitor cell-mediated gene therapy Revised recommendations for the management of Gaucher disease in children.Multi-system disorders of glycosphingolipid and ganglioside metabolism A symptomatic Fabry disease mouse model generated by inducing globotriaosylceramide synthesis.Glucocerebrosidase gene-deficient mouse recapitulates Gaucher disease displaying cellular and molecular dysregulation beyond the macrophage.Gaucher disease and its treatment options.High incidence of cholesterol gallstone disease in type 1 Gaucher disease: characterizing the biliary phenotype of type 1 Gaucher disease.Gaucher disease: clinical profile and therapeutic developments.Neuronopathic Gaucher's disease: induced pluripotent stem cells for disease modelling and testing chaperone activity of small compounds.Gaucher disease: new developments in treatment and etiology.Expression of human Gaucher disease gene GBA generates neurodevelopmental defects and ER stress in Drosophila eye A prospective study of bone marrow hematopoietic and mesenchymal stem cells in type 1 Gaucher disease patients Gaucher Disease-Induced Pluripotent Stem Cells Display Decreased Erythroid Potential and Aberrant Myelopoiesis.Gaucher disease gene GBA functions in immune regulation.Elevation of glycoprotein nonmetastatic melanoma protein B in type 1 Gaucher disease patients and mouse models Glucosylated cholesterol in mammalian cells and tissues: formation and degradation by multiple cellular β-glucosidases.Pharmacotherapeutic strategies using small molecules for the treatment of glycolipid lysosomal storage disorders.Improved management of lysosomal glucosylceramide levels in a mouse model of type 1 Gaucher disease using enzyme and substrate reduction therapy Timing of initiation of enzyme replacement therapy after diagnosis of type 1 Gaucher disease: effect on incidence of avascular necrosis.Enzyme therapy for the treatment of type 1 Gaucher disease: clinical outcomes and dose - response relationships.Quality control gone wrong: mitochondria, lysosomal storage disorders and neurodegeneration.Glucocerebrosidase 2 gene deletion rescues type 1 Gaucher disease.Lysosomal lipid storage diseases.Animal models for Gaucher disease research.Lentiviral hematopoietic stem cell gene therapy in inherited metabolic disorders.Emerging therapeutic targets for Gaucher disease.Gaucher disease: Progress and ongoing challenges.Lentiviral gene therapy using cellular promoters cures type 1 Gaucher disease in mice.Induced Pluripotent Stem Cell Modeling of Gaucher's Disease: What Have We Learned?Glucocerebrosidase deficiency in dopaminergic neurons induces microglial activation without neurodegeneration.Functional and genetic characterization of the non-lysosomal glucosylceramidase 2 as a modifier for Gaucher disease.Gaucher disease in sheep.Glucosylsphingosine Causes Hematological and Visceral Changes in Mice-Evidence for a Pathophysiological Role in Gaucher Disease.B cell lymphoma and myeloma in murine Gaucher's disease.The contribution of mutant GBA to the development of Parkinson disease in Drosophila.Gaucher disease

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P2860

Effective cell and gene therapy in a murine model of Gaucher disease.

http://www.wikidata.org/entity/Q33256447

description

2006 nî lūn-bûn

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2006 թուականի Սեպտեմբերին հրատարակուած գիտական յօդուած

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2006 թվականի սեպտեմբերին հրատարակված գիտական հոդված

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2006年の論文

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2006年論文

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2006年論文

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2006年論文

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2006年論文

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2006年論文

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2006年论文

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name

Effective cell and gene therapy in a murine model of Gaucher disease.

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Effective cell and gene therapy in a murine model of Gaucher disease.

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Effective cell and gene therapy in a murine model of Gaucher disease.

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type

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Effective cell and gene therapy in a murine model of Gaucher disease.

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Effective cell and gene therapy in a murine model of Gaucher disease.

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Effective cell and gene therapy in a murine model of Gaucher disease.

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Effective cell and gene therapy in a murine model of Gaucher disease.

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Effective cell and gene therapy in a murine model of Gaucher disease.

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Effective cell and gene therapy in a murine model of Gaucher disease.

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PNAS.0606016103

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Proceedings of the National Academy of Sciences of the United States of America

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Effective cell and gene therapy in a murine model of Gaucher disease.

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Andreas Ooka

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Eva Nilsson

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Ida Berglin Enquist

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Jan-Eric Månsson

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Johan Richter

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Karin Olsson

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Mats Ehinger

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Roscoe O Brady

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Stefan Karlsson

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P2860

Metaxin, a gene contiguous to both thrombospondin 3 and glucocerebrosidase, is required for embryonic development in the mouse: implications for Gaucher disease

Enzyme replacement in Gaucher disease

Gene therapy of human severe combined immunodeficiency (SCID)-X1 disease

Gaucher disease: new molecular approaches to diagnosis and treatment

Consequences of beta-glucocerebrosidase deficiency in epidermis. Ultrastructure and permeability barrier alterations in Gaucher disease

Mice with type 2 and 3 Gaucher disease point mutations generated by a single insertion mutagenesis procedure

Inducible gene targeting in mice

TGF-beta signaling-deficient hematopoietic stem cells have normal self-renewal and regenerative ability in vivo despite increased proliferative capacity in vitro.

Effectiveness of enzyme replacement therapy in 1028 patients with type 1 Gaucher disease after 2 to 5 years of treatment: a report from the Gaucher Registry.

Demonstration of a deficiency of glucocerebroside-cleaving enzyme in Gaucher's disease.

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