Single-Cell Sequencing of iPSC-Dopamine Neurons Reconstructs Disease Progression and Identifies HDAC4 as a Regulator of Parkinson Cell Phenotypes

Bulk and single-cell RNA-seq of iPSC-derived dopamine neurons from control and PD GBA-N370S patients stratified a clinically distinct patient and revealed HDAC4 as a potential therapeutic PD target. Pharmacological modulation of HDAC4 rescued PD-related phenotypes in GBA-N370S neurons. HDAC4 perturbation was also observed in a subset of sporadic PD patients.

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