MicroRNAs-424 and 206 are potential prognostic markers in spinal onset amyotrophic lateral sclerosis

•MicroRNAs hsa-miR-424 and 206 are overexpressed in the skeletal muscle of patients with ALS.•MicroRNAs hsa-miR-424 and 206 are overexpressed in the serum of patients with ALS.•Baseline serum expression of these microRNAs correlates with ALS progression.

IntroductionSkeletal muscle microRNAs (miRNAs) are potential candidate biomarkers for amyotrophic lateral sclerosis (ALS) that deserve further investigation.ObjectivesTo identify miRNAs abnormally expressed in the skeletal muscle and plasma of patients with ALS, and to correlate them with parameters of disease progression.MethodsExpression profile of miRNAs in muscle was evaluated using an array platform. Subsequently we assessed the plasmatic expression of candidate miRNAs in a set of 39 patients/39 controls. We employed generalized estimating equations to investigate correlations with clinical data.ResultsWe identified 11 miRNAs differentially expressed in the muscle of ALS patients; of these, miR424, miR-214 and miR-206 were validated by qPCR in muscle samples. In plasma, we found only miR-424 and miR 206 to be overexpressed. Baseline expression of miR-424 and 206 correlated with clinical deterioration over time.ConclusionMiR-424 and miR-206 are potential prognostic markers for ALS.