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

by 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
Published 2021-11-01

Decreased glucocerebrosidase activity and substrate accumulation of glycosphingolipids in a novel GBA1 D409V knock-in mouse model.

by Nicole K Polinski, Terina N Martinez, Alexander Gorodinsky, Ralph Gareus, Michael Sasner, Mark Herberth, Robert Switzer, Syed O Ahmad, Mali Cosden, Monika Kandebo, Robert E Drolet, Peter D Buckett, Weisong Shan, Yi Chen, Lee J Pellegrino, Gregory D Ellsworth, Leo B Dungan, Warren D Hirst, Sean W Clark, Kuldip D Dave
Published 2021-01-01