Enzyme replacement improves ataxic gait and central nervous system histopathology in a mouse model of metachromatic leukodystrophy. - Test2 - elhamkhani - TriplyDB

Enzyme replacement improves ataxic gait and central nervous system histopathology in a mouse model of metachromatic leukodystrophy.

Enzyme replacement improves ataxic gait and central nervous system histopathology in a mouse model of metachromatic leukodystrophy.

http://www.wikidata.org/entity/Q39505508

about

Developing therapeutic approaches for metachromatic leukodystrophy Cerebellar alterations and gait defects as therapeutic outcome measures for enzyme replacement therapy in α-mannosidosis.Multi-system disorders of glycosphingolipid and ganglioside metabolism Correction of CNS defects in the MPSII mouse model via systemic enzyme replacement therapy.Lysosomal storage disease: gene therapy on both sides of the blood-brain barrier.Correction of brain oligodendrocytes by AAVrh.10 intracerebral gene therapy in metachromatic leukodystrophy mice.Effect of systemic high dose enzyme replacement therapy on the improvement of CNS defects in a mouse model of mucopolysaccharidosis type II Chronic enzyme replacement therapy ameliorates neuropathology in alpha-mannosidosis mice Arylsulfatase A Overexpressing Human iPSC-derived Neural Cells Reduce CNS Sulfatide Storage in a Mouse Model of Metachromatic Leukodystrophy.Direct tandem mass spectrometric profiling of sulfatides in dry urinary samples for screening of metachromatic leukodystrophy.Myelin lesions associated with lysosomal and peroxisomal disorders.Emerging therapies for neurodegenerative lysosomal storage disorders - from concept to reality.Childhood leukodystrophies: a clinical perspective.Blood-brain barrier structure and function and the challenges for CNS drug delivery.Gene therapy for the neurological manifestations in lysosomal storage disorders.Outcome of Early Juvenile Onset Metachromatic Leukodystrophy After Unrelated Cord Blood Transplantation: A Case Series and Review of the Literature.Gene therapy for lysosomal storage disorders: recent advances for metachromatic leukodystrophy and mucopolysaccaridosis I.High-dose enzyme replacement therapy in murine Hurler syndrome Identification of 83 novel alpha-mannosidosis-associated sequence variants: functional analysis of MAN2B1 missense mutations.IDS crossing of the blood-brain barrier corrects CNS defects in MPSII mice.Transport of arylsulfatase A across the blood-brain barrier in vitro.Long-term correction of biochemical and neurological abnormalities in MLD mice model by neonatal systemic injection of an AAV serotype 9 vector.Promising CNS-directed enzyme replacement therapy for lysosomal storage diseases.Complete mapping of a cystine knot and nested disulfides of recombinant human arylsulfatase A by multi-enzyme digestion and LC-MS analysis using CID and ETD.Efficacy of enzyme replacement therapy in an aggravated mouse model of metachromatic leukodystrophy declines with age.Alpha-Mannosidosis: Therapeutic Strategies.Nonclinical comparability studies of recombinant human arylsulfatase A addressing manufacturing process changes.

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P2860

Enzyme replacement improves ataxic gait and central nervous system histopathology in a mouse model of metachromatic leukodystrophy.

http://www.wikidata.org/entity/Q39505508

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2009 nî lūn-bûn

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2009年の論文

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2009年学术文章

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2009年学术文章

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2009年学术文章

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2009年学术文章

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2009年學術文章

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2009年學術文章

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2009年學術文章

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name

Enzyme replacement improves at ...... metachromatic leukodystrophy.

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Enzyme replacement improves at ...... metachromatic leukodystrophy.

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Enzyme replacement improves at ...... metachromatic leukodystrophy.

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Enzyme replacement improves at ...... metachromatic leukodystrophy.

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Enzyme replacement improves at ...... metachromatic leukodystrophy.

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Molecular Therapy

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Enzyme replacement improves at ...... metachromatic leukodystrophy.

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Carl Eistrup

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Cecilia Weigelt

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Claes Andersson

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Jens Fogh

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Stijn Stroobants

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Ulrich Matzner

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Volkmar Gieselmann

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P2860

Enzyme replacement improves nervous system pathology and function in a mouse model for metachromatic leukodystrophy

Enzyme replacement therapy in a mouse model of aspartylglycosaminuria

The biogenesis of lysosomes

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

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

Enzyme-replacement therapy for metabolic storage disorders.

Overcoming the blood-brain barrier with high-dose enzyme replacement therapy in murine mucopolysaccharidosis VII.

Efficacy of enzyme replacement therapy in alpha-mannosidosis mice: a preclinical animal study.

Gene therapy of metachromatic leukodystrophy reverses neurological damage and deficits in mice.

Neurobiology and cellular pathogenesis of glycolipid storage diseases.

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600-606

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10.1038/MT.2008.305

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4

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17

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Renate Lüllmann-Rauch

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23954124

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