Modest phenotypic improvements in ASA-deficient mice with only one UDP-galactose: ceramide-galactosyltransferase gene

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De Deyn PP; D'Hooge R; Mansson JE; Wittke D; Franken S; Lüllmann-Rauch R; Matzner U; Gieselmann V;

<p>Summary</p> <p>Background</p> <p>Arylsulfatase A (ASA)-deficient mice are a model for the lysosomal storage disorder metachromatic leukodystrophy. This lipidosis is characterised by the lysosomal accumulation of the sphingolipid sulfatide. Storage of this lipid is as... View more
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