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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Publicado no:Sci Rep
Main Authors: 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.
Formato: Artigo
Idioma:Inglês
Publicado em: Nature Publishing Group UK 2021
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Article
Acesso em linha: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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