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

7 May 2025

Abstract excerpt

Biallelic mutations in the glucosylceramidase beta 1 (GBA1) gene are the underlying genetic cause of Gaucher's disease (GD), resulting in a deficient lysosomal hydrolase and subsequent accumulation of glycosphingolipids. More recently, GBA1 mutations have been identified as the most prevalent genetic risk factor for Parkinson's disease (PD), associated with more pronounced symptoms characterized by earlier onset...

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PMID: 40333681
DOI: 10.1371/journal.pone.0321145

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