Druggable transcriptomic pathways revealed in Parkinson's patient-derived midbrain neurons.

ABSTRACT: Complex genetic predispositions accelerate the chronic degeneration of midbrain substantia nigra neurons in Parkinson's disease (PD). Deciphering the human molecular makeup of PD pathophysiology can guide the discovery of therapeutics to slow the disease progression. However, insights from human postmortem brain studies only portray the latter stages of PD, and there is a lack of data surrounding molecular events preceding the neuronal loss in patients. We address this gap by identifying the gene dysregulation of live midbrain neurons reprogrammed in vitro from the skin cells of 42 individuals, including sporadic and familial PD patients and matched healthy controls. To minimize bias resulting from neuronal reprogramming and RNA-seq methods, we developed an analysis pipeline integrating PD

SUBMITTER: van den Hurk M 

PROVIDER: S-EPMC9579158 | biostudies-literature | 2022 Oct

REPOSITORIES: biostudies-literature