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Article

Gene Therapy for Parkinson's Disease Associated with GBA1 Mutations.

Journal of Parkinson's disease - 1 Jan 2000

Abeliovich Asa, Hefti Franz, Sevigny Jeffrey

Abstract excerpt

Human genetic studies as well as studies in animal models indicate that lysosomal dysfunction plays a key role in the pathogenesis of Parkinson's disease. Among the lysosomal genes involved, GBA1 has the largest impact on Parkinson's disease risk. Deficiency in the GBA1 encoded enzyme glucocerebrosidase (GCase) leads to the accumulation of the GCase glycolipid substrates glucosylceramide and glucosylsphingosine...

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Identifiers

PMID: 34151863
DOI: 10.3233/JPD-212739
PMCID: PMC8543272

Provenance

PubMed metadata and Literature Corpus relationships. Corpus snapshot updated 7 Sept 2026. Manifest publication-graph-cohort-2026-09-07.1.

Source receipt: pubmed, retrieved 7 Sept 2026.

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