Regulated microRNAs in the CSF of patients with multiple sclerosis A case-control study

ABSTRACT Objective: MicroRNAs (miRNAs) are small noncoding RNAs that regulate gene expression and serve as promising therapeutic targets in many diseases. MiRNAs are also present in biological fluids and may be of use as disease biomarkers. We evaluated whether miRNAs are differentially regulated in the CSF of patients with multiple sclerosis (MS). Methods: The CSF of 53 patients with MS and 39 patients with other neurologic diseases (OND) was analyzed. First, global miRNA profiling was assessed to screen for reported miRNAs, followed by quantitative reverse transcriptase PCR to validate candidate miRNAs. Results: After global miRNA profiling, we quantitatively confirmed miR-922 ( p = 0.0001), miR-181c ( p = 0.0007), and miR-633 ( p = 0.0014) to be differentially regulated in patients with MS as compared with OND. Importantly, miR-181c and miR-633 differentiated relapsing-remitting from secondary progressive MS courses with specificity up to of 82% and a sensitivity of 69%. Conclusion: CSF-based miRNAs were differentially regulated in patients with MS as compared with OND and in different MS disease courses. Despite the preliminary character of our case-control study, the results provide rationale for a confirmation study in larger MS cohorts.