Reduction of globotriaosylceramide in Fabry disease mice by substrate deprivation

We used a potent inhibitor of glucosylceramide synthase to test whether substrate deprivation could lower globotriaosylceramide levels in α-galactosidase A (α-gal A) knockout mice, a model of Fabry disease. C57BL/6 mice treated twice daily for 3 days with D-threo-1-ethylendioxyphenyl-2-palmitoylamin...

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Hauptverfasser: Abe, Akira, Gregory, Susan, Lee, Lihsueh, Killen, Paul D., Brady, Roscoe O., Kulkarni, Ashok, Shayman, James A.
Format: Artigo
Sprache:Inglês
Veröffentlicht: American Society for Clinical Investigation 2000
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Article
Online Zugang:https://ncbi.nlm.nih.gov/pmc/articles/PMC300859/
https://ncbi.nlm.nih.gov/pubmed/10841515
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