A New Glucocerebrosidase Chaperone Reduces α-Synuclein and Glycolipid Levels in iPSC-Derived Dopaminergic Neurons from Patients with Gaucher Disease and Parkinsonism.

doi:10.1523/JNEUROSCI.0636-16.2016

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Article

A New Glucocerebrosidase Chaperone Reduces α-Synuclein and Glycolipid Levels in iPSC-Derived Dopaminergic Neurons from Patients with Gaucher Disease and Parkinsonism.

The Journal of neuroscience : the official journal of the Society for Neuroscience - 13 Jul 2016

Aflaki Elma, Borger Daniel K, Moaven Nima, Stubblefield Barbara K, Rogers Steven A, Patnaik Samarjit, Schoenen Frank J, Westbroek Wendy, Zheng Wei, Sullivan Patricia, Fujiwara Hideji, Sidhu Rohini, Khaliq Zayd M, Lopez Grisel J, Goldstein David S, Ory Daniel S, Marugan Juan, Sidransky Ellen

Abstract excerpt

UNLABELLED: Among the known genetic risk factors for Parkinson disease, mutations in GBA1, the gene responsible for the lysosomal disorder Gaucher disease, are the most common. This genetic link has directed attention to the role of the lysosome in the pathogenesis of parkinsonism. To study how glucocerebrosidase impacts parkinsonism and to evaluate new therapeutics, we generated induced human pluripotent stem...

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Identifiers

PMID: 27413154
DOI: 10.1523/JNEUROSCI.0636-16.2016
PMCID: PMC4945664

Genes

GBA1

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PubMed metadata and Literature Corpus relationships. Corpus snapshot updated 7 Sept 2026. Manifest publication-graph-cohort-2026-09-08.1.

Source receipt: pubmed, retrieved 7 Sept 2026.

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