PGRN deficiency exacerbates, whereas a brain penetrant PGRN derivative protects, GBA1 mutation-associated pathologies and diseases

Mutations in , encoding glucocerebrosidase (GCase), cause Gaucher disease (GD) and are also genetic risks in developing Parkinson's disease (PD). Currently, the approved therapies are only effective for directly treating visceral symptoms, but not for primary neuronopathic involvement in GD (nG...

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Bibliographic Details
Published in:	Proceedings of the National Academy of Sciences - PNAS Vol. 120; no. 1; p. e2210442120
Main Authors:	Zhao, Xiangli, Lin, Yi, Liou, Benjamin, Fu, Wenyu, Jian, Jinlong, Fannie, Venette, Zhang, Wujuan, Setchell, Kenneth D R, Grabowski, Gregory A, Sun, Ying, Liu, Chuan-Ju
Format:	Journal Article
Language:	English
Published:	United States National Academy of Sciences 03-01-2023
Subjects:	Animals Autophagy Biological Sciences Blood-brain barrier Brain - metabolism Fibroblasts Gaucher Disease - genetics Gaucher's disease Gliosis Glucosylceramidase Glucosylceramidase - genetics Glucosylceramidase - metabolism In vivo methods and tests Inflammation Lysosomal storage diseases Lysosomes - metabolism Mice Mice, Knockout Movement disorders Mutation Neurodegenerative diseases Parkinson Disease - genetics Parkinson's disease Phenotypes Progranulins - genetics Signs and symptoms progranulin Gaucher disease progranulin-derived biologic Parkinson’s disease GBA1 mutation
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