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Article

AAV gene therapy for GBA1-related diseases.

Molecular therapy : the journal of the American Society of Gene Therapy - 4 Mar 2026

Ayloo Swathi, Ryu Jae Cheon, Chou Shih-Ching, Blatnik Lydia, Wischhof Erik, Bu Jie, Guo Lilu, Hossain Mahmud, Ghosh Dhiman, McCarty William, Byrne Ann, Chai Lilly, Clarke Jennifer, Kayatekin Can, Zhang Bailin, Sardi S Pablo, Goulet Martin, Elmer Bradford, Mueller Christian, Ramachandran Shyam

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,...

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Source

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Identifiers

PMID: 41376160
DOI: 10.1016/j.ymthe.2025.12.011
PMCID: PMC12974211

Genes

GBA1

Provenance

PubMed metadata and Literature Corpus relationships. Corpus snapshot updated 1 Sept 2026.

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