Bone marrow transplantation increases efficacy of central nervous system-directed enzyme replacement therapy in the murine model of globoid cell leukodystrophy - Wikidata - awgldk - TriplyDB

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

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

http://www.wikidata.org/entity/Q36240279

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New therapeutic approaches for Krabbe disease: The potential of pharmacological chaperones Lysosomal Re-acidification Prevents Lysosphingolipid-Induced Lysosomal Impairment and Cellular Toxicity Combined gene/cell therapies provide long-term and pervasive rescue of multiple pathological symptoms in a murine model of globoid cell leukodystrophy.Therapeutic benefit of lentiviral-mediated neonatal intracerebral gene therapy in a mouse model of globoid cell leukodystrophy On the role of natural killer cells in neurodegenerative diseases Experimental therapies in the murine model of globoid cell leukodystrophy.Gene therapy for lysosomal storage disorders: a good start.Mechanism-based combination treatment dramatically increases therapeutic efficacy in murine globoid cell leukodystrophy Azasugar inhibitors as pharmacological chaperones for Krabbe disease Neuroinflammatory paradigms in lysosomal storage diseases.Long-term Improvements in Lifespan and Pathology in CNS and PNS After BMT Plus One Intravenous Injection of AAVrh10-GALC in Twitcher Mice Multipotent stromal cells alleviate inflammation, neuropathology, and symptoms associated with globoid cell leukodystrophy in the twitcher mouse.Cellular transplant therapies for globoid cell leukodystrophy: Preclinical and clinical observations Combination Therapies for Lysosomal Storage Diseases: A Complex Answer to a Simple Problem.Krabbe disease: One Hundred years from the bedside to the bench to the bedside.Treatment for Krabbe's disease: Finding the combination.Chaperones as potential therapeutics for Krabbe disease.Perspective on innovative therapies for globoid cell leukodystrophy.Immunological considerations for treating globoid cell leukodystrophy.A HILIC-MS/MS method for simultaneous quantification of the lysosomal disease markers galactosylsphingosine and glucosylsphingosine in mouse serum

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P2860

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

http://www.wikidata.org/entity/Q36240279

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J.YMGME.2012.05.021

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Molecular Genetics and Metabolism

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Bone marrow transplantation in ...... of globoid cell leukodystrophy

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P2093

Adarsh S Reddy

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Daniel S Ory

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Elizabeth Y Qin

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Jacqueline A Hawkins-Salsbury

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Nuri B Farber

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Xuntian Jiang

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P2860

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

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

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

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

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

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

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

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.

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

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

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186-196

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10.1016/J.YMGME.2012.05.021

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1-2

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107

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Enzyme replacement therapy

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3444533

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