AAV delivery of GBA1 suppresses α-synuclein accumulation in Parkinson's disease models and restores functions in Gaucher's disease models.

PLoS One · 2025 · PMC12057913 · PMID 40333681

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highly significantno p-value reported

Even though we observed an increase of GCase activity above wild-type level ( S1 Table ), GlcSph reduction (3 ~ 4 folds) ( S2 Table ), while highly significant, did not reach the wild-type level most likely due to AAV vector biodistribution and cell type tropism.

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