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Royal Society of Chemistry, MedChemComm, 1(5), p. 68-71

DOI: 10.1039/c3md00257h

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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

This paper is available in a repository.
This paper is available in a repository.

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Abstract

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.