As for Duchenne muscular dystrophy [27], [28], quantification of selected miRNAs might be used as a sensitive biomarker tool of CF severity and functional suppression of CFTR-targeting miRNAs, such as miR-101 and/or miR-494, could prove a strategy to efficiently restore CFTR synthesis in patients carrying mutations leading to insufficient protein expression. The gene discussed is CFTR; the disease is cystic fibrosis.