Profiling urine miRNAs in laminin alpha 2 chain-deficient muscular dystrophy as potential biomarkers

Circulating miRNAs are being considered as non-invasive biomarkers for disease progression and clinical trials. Congenital muscular dystrophy with deficiency of laminin a2 chain is a very severe form of muscular dystrophy, for which no treatment is available. In order to identify MDC1A biomarkers we have profiled miRNA in urine from MDC1A mice at three distinct time points (asymptomatic, initial symptoms and established disease). We demonstrate that unique groups of miRNAs are differentially expressed at each time point. To our knowledge, this is the first time that a urine microRNA profile for muscular dystrophy has been presented. We suggest that urine miRNAs can be sensitive biomarkers for different stages of MDC1A.