Arylsulfatase A Overexpressing Human iPSC-derived Neural Cells Reduce CNS Sulfatide Storage in a Mouse Model of Metachromatic Leukodystrophy

Metachromatic leukodystrophy (MLD) is an inherited lysosomal storage disorder resulting from a functional deficiency of arylsulfatase A (ARSA), an enzyme that catalyzes desulfation of 3-O-sulfogalactosylceramide (sulfatide). Lack of active ARSA leads to the accumulation of sulfatide in oligodendrocy...

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Bibliographic Details
Published in:Mol Ther
Main Authors: Doerr, Jonas, Böckenhoff, Annika, Ewald, Benjamin, Ladewig, Julia, Eckhardt, Matthias, Gieselmann, Volkmar, Matzner, Ulrich, Brüstle, Oliver, Koch, Philipp
Format: Artigo
Language:Inglês
Published: Nature Publishing Group 2015
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Original Article
Online Access:https://ncbi.nlm.nih.gov/pmc/articles/PMC4817891/
https://ncbi.nlm.nih.gov/pubmed/26061647
https://ncbi.nlm.nih.govhttp://dx.doi.org/10.1038/mt.2015.106
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