Enzyme Replacement Improves Ataxic Gait and Central Nervous System Histopathology in a Mouse Model of Metachromatic Leukodystrophy

Inherited deficiencies of lysosomal hydrolases cause lysosomal storage diseases (LSDs) that are characterized by a progressive multisystemic pathology and premature death. Repeated intravenous injection of the active counterpart of the deficient enzyme, a treatment strategy called enzyme replacement...

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Main Authors: Matzner, Ulrich, Lüllmann-Rauch, Renate, Stroobants, Stijn, Andersson, Claes, Weigelt, Cecilia, Eistrup, Carl, Fogh, Jens, D'Hooge, Rudi, Gieselmann, Volkmar
Formato: Artigo
Idioma:Inglês
Publicado: Nature Publishing Group 2009
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Original Articles
Acceso en liña:https://ncbi.nlm.nih.gov/pmc/articles/PMC2835113/
https://ncbi.nlm.nih.gov/pubmed/19174759
https://ncbi.nlm.nih.govhttp://dx.doi.org/10.1038/mt.2008.305
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