Design, synthesis and biochemical investigation, by in vitro luciferase reporter system, of peptide nucleic acids as new inhibitors of miR-509-3p involved in the regulation of cystic fibrosis disease-gene expression

Recently, microRNAs (miRNAs) were identified as being able to inhibit the expression of the Cystic Fibrosis Transmembrane Regulator (CFTR) disease-gene of Cystic Fibrosis (CF) and CFTR-Related Disorders (CFTR-RD). This study shows the use of peptide nucleic acids (PNAs) as inhibitors of miR-509-3p, one of the CFTR regulating miRNAs, by reverting the expression of the luciferase gene containing the 3′UTR of CFTR gene.