New Molecular Therapeutic Approaches for Cystic Fibrosis

Dear Colleagues,

Lung disease represents the main cause of cystic fibrosis (CF) morbidity and mortality. In the bronchial epithelium, a dysfunction of cystic fibrosis transmembrane conductance regulator (CFTR) causes the accumulation of dense mucus, chronic infection, and neutrophilic inflammation, which can lead to respiratory insufficiency and death. The advent of CFTR modulators has improved the quality of life of many people with CF. However, although F508-del is the most frequent mutation, effective therapy for most of disease-causing mutations identified in CF is not yet available. Moreover, recurrent pathogen infections reduce modulator efficacy and sustain a chronic inflammatory status still requiring treatment with antibacterial agents.

Molecular therapeutics have been developed in the recent years and they could be exploited in CF to correct mutations, to reduce inflammation and to develop antibacterial drugs.

A main problem in CF research is the need of adequate ex vivo and preclinical models, and the development of respiratory 3D structures, as organoids and lungs/airways-on-chip, has provided new tools for studying lung diseases, and could be suitable for new therapeutic approches in CF.

This Special Issue aims to be a call for original articles/reviews about molecular therapeutic approaches in CF regarding:

• CFTR correction/modulation/overexpression;
• Transfection of differentiated epithelia;
• Pathogen infection/antibiotic resistance;
• Techniques of drug delivery;
• 3D models suitable for drug testing.

Dr. Roberto Plebani
Dr. Onofrio Laselva
Guest Editors

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• cystic fibrosis
• inflammation
• 3D structures
• organ-on-chip technology
• organoids
• endothelium
• pulmonary disease
• pathogens
• drug resistance
• miRNA
• RNA interfering
• CRISPR/Cas9
• transfection
• drug delivery