Substrate reduction therapy for Krabbe disease and metachromatic leukodystrophy using a novel ceramide galactosyltransferase inhibitor

Krabbe disease (KD) and metachromatic leukodystrophy (MLD) are caused by accumulation of the glycolipids galactosylceramide (GalCer) and sulfatide and their toxic metabolites psychosine and lysosulfatide, respectively. We discovered a potent and selective small molecule inhibitor (S202) of ceramide...

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Veröffentlicht in:Sci Rep
Hauptverfasser: Babcock, Michael C., Mikulka, Christina R., Wang, Bing, Chandriani, Sanjay, Chandra, Sundeep, Xu, Yue, Webster, Katherine, Feng, Ying, Nelvagal, Hemanth R., Giaramita, Alex, Yip, Bryan K., Lo, Melanie, Jiang, Xuntian, Chao, Qi, Woloszynek, Josh C., Shen, Yuqiao, Bhagwat, Shripad, Sands, Mark S., Crawford, Brett E.
Format: Artigo
Sprache:Inglês
Veröffentlicht: Nature Publishing Group UK 2021
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Article
Online Zugang:https://ncbi.nlm.nih.gov/pmc/articles/PMC8280112/
https://ncbi.nlm.nih.gov/pubmed/34262084
https://ncbi.nlm.nih.govhttp://dx.doi.org/10.1038/s41598-021-93601-1
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