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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Опубликовано в: :Sci Rep
Главные авторы: 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.
Формат: Artigo
Язык:Inglês
Опубликовано: Nature Publishing Group UK 2021
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Article
Online-ссылка: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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