Enzyme replacement improves ataxic gait and central nervous system histopathology in a mouse model of metachromatic leukodystrophy.
about
Developing therapeutic approaches for metachromatic leukodystrophyCerebellar alterations and gait defects as therapeutic outcome measures for enzyme replacement therapy in α-mannosidosis.Multi-system disorders of glycosphingolipid and ganglioside metabolismCorrection 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 IIChronic enzyme replacement therapy ameliorates neuropathology in alpha-mannosidosis miceArylsulfatase 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 syndromeIdentification 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.
P2860
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P2860
Enzyme replacement improves ataxic gait and central nervous system histopathology in a mouse model of metachromatic leukodystrophy.
description
2009 nî lūn-bûn
@nan
2009年の論文
@ja
2009年学术文章
@wuu
2009年学术文章
@zh-cn
2009年学术文章
@zh-hans
2009年学术文章
@zh-my
2009年学术文章
@zh-sg
2009年學術文章
@yue
2009年學術文章
@zh
2009年學術文章
@zh-hant
name
Enzyme replacement improves at ...... metachromatic leukodystrophy.
@en
Enzyme replacement improves at ...... metachromatic leukodystrophy.
@nl
type
label
Enzyme replacement improves at ...... metachromatic leukodystrophy.
@en
Enzyme replacement improves at ...... metachromatic leukodystrophy.
@nl
prefLabel
Enzyme replacement improves at ...... metachromatic leukodystrophy.
@en
Enzyme replacement improves at ...... metachromatic leukodystrophy.
@nl
P2093
P2860
P356
P1433
P1476
Enzyme replacement improves at ...... metachromatic leukodystrophy.
@en
P2093
Carl Eistrup
Cecilia Weigelt
Claes Andersson
Renate Lüllmann-Rauch
Rudi D'Hooge
Stijn Stroobants
Ulrich Matzner
Volkmar Gieselmann
P2860
P304
P356
10.1038/MT.2008.305
P577
2009-01-27T00:00:00Z