Comparative study of enriched dopaminergic neurons from siblings with Gaucher disease discordant for parkinsonism.

ABSTRACT: Inducible pluripotent stem cells (iPSCs) derived from patient samples have significantly enhanced our ability to model neurological diseases. Comparative studies of dopaminergic (DA) neurons differentiated from iPSCs derived from siblings with Gaucher disease discordant for parkinsonism provides a valuable avenue to explore genetic modifiers contributing to GBA1-associated parkinsonism in disease-relevant cells. However, such studies are often complicated by the inherent heterogeneity in differentiation efficiency among iPSC lines derived from different individuals. To address this technical challenge, we devised a selection strategy to enrich dopaminergic (DA) neurons expressing tyrosine hydroxylase (TH). A neomycin resistance gene (neo) was inserted at the C-terminus of the

SUBMITTER: Hertz E 

PROVIDER: S-EPMC10962709 | biostudies-literature | 2024 Feb

REPOSITORIES: biostudies-literature