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...
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 |
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Format: | Article |
Language: | English |
Published: |
Elsevier
2021-11-01
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Series: | Neurobiology of Disease |
Subjects: | |
Online Access: | http://www.sciencedirect.com/science/article/pii/S0969996121002564 |
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