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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Detalles Bibliográficos
Autores principales: 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: Online Artículo Texto
Lenguaje:English
Publicado: Nature Publishing Group UK 2021
Materias:
Article
Acceso en línea:https://www.ncbi.nlm.nih.gov/pmc/articles/PMC8280112/
https://www.ncbi.nlm.nih.gov/pubmed/34262084
http://dx.doi.org/10.1038/s41598-021-93601-1

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