Article

AAV gene therapy for GBA-PD and Gaucher Disease

2025-06-17

Abstract excerpt

Mutations in GBA1 , the gene encoding glucocerebrosidase (GCase), are the most common risk factor for Parkinson’s Disease (PD). GBA-PD patients are a genetic subpopulation of PD carrying heterozygous mutations in GBA1 . Additionally, bi-allelic mutations in GBA1 cause Gaucher Disease (GD), a lysosomal storage disorder. Loss of GCase activity, a lysosomal enzyme leads to the accumulation of lipid substrates, dis...

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Literature Corpus work: e0683858-e20c-5a0a-9c81-63ad3747f7b8
DOI: 10.1101/2025.06.17.660133

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