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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Vydáno v:Sci Rep
Hlavní autoři: 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.
Médium: Artigo
Jazyk:Inglês
Vydáno: Nature Publishing Group UK 2021
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On-line přístup: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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