A novel glucosylceramide synthase inhibitor attenuates alpha synuclein pathology and lysosomal dysfunction in preclinical models of synucleinopathy

A novel glucosylceramide synthase inhibitor attenuates alpha synuclein pathology and lysosomal dysfunction in preclinical models of synucleinopathy

Mutations in the lysosomal enzyme glucocerebrosidase (GCase, GBA1 gene) are the most common genetic risk factor for developing Parkinson's disease (PD). GCase metabolizes the glycosphingolipids glucosylceramide (GlcCer) and glucosylsphingosine (GlcSph). Mutations in GBA1 reduce enzyme activity...

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Bibliographic Details
Main Authors: Mali Cosden, Sarah Jinn, Lihang Yao, Cheryl A. Gretzula, Monika Kandebo, Dawn Toolan, Nathan G. Hatcher, Lei Ma, Wei Lemaire, Gregory C. Adam, Christine Burlein, Christina Minnick, Rose Flick, Marla L. Watt, James Mulhearn, Mark Fraley, Robert E. Drolet, Jacob N. Marcus, Sean M. Smith
Format: Article
Language:English
Published: Elsevier 2021-11-01
Series:Neurobiology of Disease
Subjects:
Glucocerebrosidase
Glucosylceramide synthase inhibitor
Glycosphingolipids
Parkinson's disease
Lysosomal biology
Preformed fibril model
Online Access:http://www.sciencedirect.com/science/article/pii/S0969996121002564

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http://www.sciencedirect.com/science/article/pii/S0969996121002564

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