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Significance This study carries immediate significance and novelty from following perspectives: (a) It provides the first line of in vivo and ex vivo evidence demonstrating the crucial role of PGRN… Click to show full abstract
Significance This study carries immediate significance and novelty from following perspectives: (a) It provides the first line of in vivo and ex vivo evidence demonstrating the crucial role of PGRN in GBA1 mutation-related diseases (i.e., GD and PD) and advances our understanding about the pathogenesis of GD and probably PD as well. (b) It also presents a clinically relevant mouse model for mechanistic and potential therapeutics studies for GBA1 mutation-caused diseases, overcoming the barriers to allow the study in multiple organs in viscera and CNS. (c) Most excitingly, it develops a brain penetrant PGRN-derived biologics that protects against GD and PD-like pathologies, providing a potential treatment for rare lysosomal storage diseases and common neurodegenerative disorders, particularly nGD and PD.
Journal Title: Proceedings of the National Academy of Sciences of the United States of America
Year Published: 2022
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