Next Article in Journal
Antifungal and Ichthyotoxic Sesquiterpenoids from Santalum album Heartwood
Next Article in Special Issue
Applications of PNA-Based Artificial Restriction DNA Cutters
Previous Article in Journal
Dereplication-Guided Isolation of New Phenylpropanoid-Substituted Diglycosides from Cistanche salsa and Their Inhibitory Activity on NO Production in Macrophage
Article Menu
Issue 7 (July) cover image

Export Article

Open AccessArticle
Molecules 2017, 22(7), 1144;

Peptide Nucleic Acids as miRNA Target Protectors for the Treatment of Cystic Fibrosis

Department of Biosciences and Territory, University of Molise, 86170 Isernia, Italy
CEINGE–Advanced Biotechnologies Scarl, 80131 Napoli, Italy
Department of Molecular Medicine and Medical Biotechnologies, University of Naples Federico II, 80131 Napoli, Italy
Department of Pharmacy, University of Naples Federico II, 80131 Napoli, Italy
Author to whom correspondence should be addressed.
Received: 31 May 2017 / Revised: 3 July 2017 / Accepted: 4 July 2017 / Published: 8 July 2017
(This article belongs to the Special Issue Molecular Properties and the Applications of Peptide Nucleic Acids)
View Full-Text   |   Download PDF [2941 KB, uploaded 8 July 2017]   |  


Cystic Fibrosis (CF) is one of the most common life shortening conditions in Caucasians. CF is caused by mutations in the CF Transmembrane Conductance Regulator (CFTR) gene which result in reduced or altered CFTR functionality. Several microRNAs (miRNAs) downregulate the expression of CFTR, thus causing or exacerbating the symptoms of CF. In this context, the design of anti-miRNA agents represents a valid functional tool, but its translation to the clinic might lead to unpredictable side effects because of the interference with the expression of other genes regulated by the same miRNAs. Herein, for the first time, is proposed the use of peptide nucleic acids (PNAs) to protect specific sequences in the 3’UTR (untranslated region) of the CFTR messenger RNA (mRNA) by action of miRNAs. Two PNAs (7 and 13 bases long) carrying the tetrapeptide Gly-SerP-SerP-Gly at their C-end, fully complementary to the 3’UTR sequence recognized by miR-509-3p, have been synthesized and the structural features of target PNA/RNA heteroduplexes have been investigated by spectroscopic and molecular dynamics studies. The co-transfection of the pLuc-CFTR-3´UTR vector with different combinations of PNAs, miR-509-3p, and controls in A549 cells demonstrated the ability of the longer PNA to rescue the luciferase activity by up to 70% of the control, thus supporting the use of suitable PNAs to counteract the reduction in the CFTR expression. View Full-Text
Keywords: cystic fibrosis; CFTR; miRNA; miRNA target protectors; miR-509-3p; peptide nucleic acid; PNA cystic fibrosis; CFTR; miRNA; miRNA target protectors; miR-509-3p; peptide nucleic acid; PNA

Figure 1

This is an open access article distributed under the Creative Commons Attribution License which permits unrestricted use, distribution, and reproduction in any medium, provided the original work is properly cited. (CC BY 4.0).

Supplementary material


Share & Cite This Article

MDPI and ACS Style

Zarrilli, F.; Amato, F.; Morgillo, C.M.; Pinto, B.; Santarpia, G.; Borbone, N.; D’Errico, S.; Catalanotti, B.; Piccialli, G.; Castaldo, G.; Oliviero, G. Peptide Nucleic Acids as miRNA Target Protectors for the Treatment of Cystic Fibrosis. Molecules 2017, 22, 1144.

Show more citation formats Show less citations formats

Note that from the first issue of 2016, MDPI journals use article numbers instead of page numbers. See further details here.

Related Articles

Article Metrics

Article Access Statistics



[Return to top]
Molecules EISSN 1420-3049 Published by MDPI AG, Basel, Switzerland RSS E-Mail Table of Contents Alert
Back to Top