Correction of metachromatic leukodystrophy in the mouse model by transplantation of genetically modified hematopoietic stem cells. - Wikidata - awgldk - TriplyDB

Correction of metachromatic leukodystrophy in the mouse model by transplantation of genetically modified hematopoietic stem cells.

Correction of metachromatic leukodystrophy in the mouse model by transplantation of genetically modified hematopoietic stem cells.

http://www.wikidata.org/entity/Q30501325

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SUMF1 enhances sulfatase activities in vivo in five sulfatase deficiencies Checkpoints to the Brain: Directing Myeloid Cell Migration to the Central Nervous System Hematopoietic stem cell gene transfer in a tumor-prone mouse model uncovers low genotoxicity of lentiviral vector integration The role and metabolism of sulfatide in the nervous system Developing therapeutic approaches for metachromatic leukodystrophy Gene therapy for metachromatic leukodystrophy.Sialic acid deposition impairs the utility of AAV9, but not peptide-modified AAVs for brain gene therapy in a mouse model of lysosomal storage disease Gene therapy augments the efficacy of hematopoietic cell transplantation and fully corrects mucopolysaccharidosis type I phenotype in the mouse model.Combined gene/cell therapies provide long-term and pervasive rescue of multiple pathological symptoms in a murine model of globoid cell leukodystrophy.Human gene therapy and imaging in neurological diseases.Expression of human factor IX gene in murine plasma through lentiviral vector-infected haematopoietic stem cells.Macrophage Ontogeny Underlies Differences in Tumor-Specific Education in Brain Malignancies.The role of microglia in human disease: therapeutic tool or target?Sulfatase modifying factor 1-mediated fibroblast growth factor signaling primes hematopoietic multilineage development.Therapeutic approaches for lysosomal storage diseases Lentiviral hematopoietic stem cell gene therapy benefits metachromatic leukodystrophy.A lentiviral gene therapy strategy for the in vitro production of feline erythropoietin.Lentivector integration sites in ependymal cells from a model of metachromatic leukodystrophy: non-B DNA as a new factor influencing integration.Gene therapy for lysosomal storage disorders: a good start.Metachromatic leukodystrophy: an overview of current and prospective treatments.Gene therapy of metachromatic leukodystrophy reverses neurological damage and deficits in mice.The galactocerebrosidase enzyme contributes to the maintenance of a functional hematopoietic stem cell niche.Neural stem cell gene therapy ameliorates pathology and function in a mouse model of globoid cell leukodystrophy.Clinical applications involving CNS gene transfer.Population control of resident and immigrant microglia by mitosis and apoptosis.CNS-directed gene therapy for lysosomal storage diseases Miglustat improves purkinje cell survival and alters microglial phenotype in feline Niemann-Pick disease type C.Lysosomal storage disorders.Mesoangioblast delivery of miniagrin ameliorates murine model of merosin-deficient congenital muscular dystrophy type 1A.Gene therapy of metachromatic leukodystrophy.Gene Therapy Corrects Mitochondrial Dysfunction in Hematopoietic Progenitor Cells and Fibroblasts from Coq9R239X Mice.Hematopoietic cell transplantation activity in Europe for inherited metabolic diseases: open issues and future directions.Correction of brain oligodendrocytes by AAVrh.10 intracerebral gene therapy in metachromatic leukodystrophy mice.Design of a regulated lentiviral vector for hematopoietic stem cell gene therapy of globoid cell leukodystrophy Astrocyte dysfunction triggers neurodegeneration in a lysosomal storage disorder.Brain conditioning is instrumental for successful microglia reconstitution following hematopoietic stem cell transplantation Current and emerging therapies for the lysosomal storage disorders.Targeted inactivation of the COP9 signalosome impairs multiple stages of T cell development.Neonatal Bone Marrow Transplantation in MPS IIIA Mice.Hematopoietic stem cell gene therapy for the multisystemic lysosomal storage disorder cystinosis.

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Correction of metachromatic leukodystrophy in the mouse model by transplantation of genetically modified hematopoietic stem cells.

http://www.wikidata.org/entity/Q30501325

description

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2004年の論文

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Journal of Clinical Investigation

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Correction of metachromatic le ...... fied hematopoietic stem cells.

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Alessandra Biffi

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Ilaria Visigalli

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Maria Sessa

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Michele De Palma

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Sergio Marchesini

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Stefano Amadio

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Ubaldo Del Carro

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P2860

Viral vectors for gene therapy: the art of turning infectious agents into vehicles of therapeutics

Synthesis, spectral properties and enzymatic hydrolysis of fluorescent derivatives of cerebroside sulfate containing long-wavelength-emission probes

Rapid response of identified resident endoneurial macrophages to nerve injury

Microglia: a sensor for pathological events in the CNS

Phenotype of arylsulfatase A-deficient mice: relationship to human metachromatic leukodystrophy

Contribution of transplanted bone marrow cells to Purkinje neurons in human adult brains.

Bone marrow transplantation for globoid cell leukodystrophy, adrenoleukodystrophy, metachromatic leukodystrophy, and Hurler syndrome.

From marrow to brain: expression of neuronal phenotypes in adult mice.

Turning blood into brain: cells bearing neuronal antigens generated in vivo from bone marrow.

Perivascular microglial cells of the CNS are bone marrow-derived and present antigen in vivo.

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Claudio Bordignon

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Angelo Quattrini

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385395

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