CSF MicroRNAs Reveal Impairment of Angiogenesis and Autophagy in Parkinson Disease.

Background And Objectives: We assessed longitudinal changes in CSF microRNAs (miRNAs) in patients with moderately severe Parkinson disease.

Methods: We used next-generation whole-genome miRNA sequencing to determine CSF miRNA expression in 75 patients with Parkinson disease after single random ascending doses of nilotinib and longitudinal miRNA expression after daily nilotinib, 150 and 300 mg, vs placebo for 1 year.

Results: Significant changes in the expression of miRNAs that control genes and pathways that regulate angiogenesis, autophagy, and the blood-brain-barrier components, primarily collagen, were observed over 1 year, suggesting impairment of these pathways in Parkinson progression in these patients.