Objective: Our objective was to identify differentially expressed, circulating miRNAs in PSP which could serve as diagnostic biomarker for this disease.

Background: Progressive supranuclear palsy (PSP) is a sporadic progressive neurodegenerative disease. The diagnosis of PSP is often challenging as it relies solely on clinical findings. Circulating microRNAs (miRNAs) have been proposed as diagnostic biomarkers in various neurodegenerative disorders including Parkinson’s disease.

Method: We performed plasma miRNA profiling by reverse transcription quantitative polymerase chain reaction array in PSP patients and age- and gender-matched, cognitively normal healthy controls. Selected, differentially expressed miRNAs were validated by qPCR. Sensitivity, specificity and area under the curve (AUC) were calculated for each of the selected miRNAs.

Results: Profiling data revealed 28 differentially expressed miRNAs in PSP group compared to control (P<0.05 with a fold change of <1.5 or >1.5). A set of five upregulated miRNAs were estimated in a cohort of 18 PSP patients and 17 healthy controls. Validation study revealed that all these five miRNAs (miR-19b-3p, miR-33a-5p, miR-130b-3p, miR-136-3p, and miR-210-3p) could be used as valuable biomarkers for identifying the disease. The sensitivity, specificity and AUC for these miRNAs were 93.33%, 88.89%, 66.67%, 73.33%, and 66.67%; 68.75%, 72.22%, 68.75%, 82.35%, and 70.59%; 0.82, 0.86, 0.78, 0.79, and 0.78 respectively.

Conclusion: Plasma miR-19b-3p, miR-33a-5p, miR-130b-3p, miR-136-3p, and miR-210-3p could be used to differentiate PSP patients from healthy controls. Large-scale studies of their biomarker potential are warranted.

References: 1. Uwatoko H, Hama Y, Iwata IT, Shirai S, Matsushima M, Yabe I, et al. Identification of plasma microRNA expression changes in multiple system atrophy and Parkinson’s disease. Mol Brain. 2019;12(1):49. 2. Marques TM, Kuiperij HB, Bruinsma IB, van Rumund A, Aerts MB, Esselink RAJ, et al. MicroRNAs in Cerebrospinal Fluid as Potential Biomarkers for Parkinson’s Disease and Multiple System Atrophy. Mol Neurobiol. 2017;54(10):7736-45. 3. Botta-Orfila T, Morató X, Compta Y, Lozano JJ, Falgàs N, Valldeoriola F, et al. Identification of blood serum micro-RNAs associated with idiopathic and LRRK2 Parkinson’s disease. J Neurosci Res. 2014;92(8):1071-7.

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MDS Abstracts - https://www.mdsabstracts.org/abstract/plasma-microrna-expression-profiling-in-patients-with-progressive-supranuclear-palsy-a-five-microrna-panel-in-plasma-was-identified-as-a-potential-biomarker-for-progressive-supranuclear-palsy/