Emerging Therapeutic Approaches for Cystic Fibrosis

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Editorial

29 November 2019

Editorial: Emerging Therapeutic Approaches for Cystic Fibrosis

Miquéias Lopes-Pacheco

,

Nicoletta Pedemonte

and

Anthony Kicic

4,846 views

7 citations

Editors

3

Miquéias Lopes-Pacheco

School of Medicine, Emory University

Nicoletta Pedemonte

Giannina Gaslini Institute (IRCCS)

Anthony Kicic

Telethon Kids Institute, University of Western Australia

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Mutations in the cystic fibrosis transmembrane conductance regulator (CFTR) gene cause cystic fibrosis (CF) – the most common life-threatening genetic disease in the Caucasian population. CFTR encodes a chloride/bicarbonate channel located at the apical membrane that regulates ionic and fluid balance across several epithelial tissues. Although CF is a multi-organ disease, respiratory failure remains the major cause of morbidity and mortality. In this line, a vicious cycle of mucus accumulation in the airways, chronic inflammation, and recurrent infections, leads to permanent tissue damage and airway remodeling, and eventually end-stage lung disease later in life.

Over the last decades, life expectancy of CF patients has lengthened, thanks to early diagnosis and more effective therapies; however, patients still face limited quality of life and significantly reduced life expectancy. Novel methods (e.g., delivery systems, high-throughput screening, precision/personalized medicine) as well as pre-clinical in vitro (e.g. organoids/spheroids, induced pluripotent stem cells, conditional reprogrammed primary airway epithelial cells) and in vivo models (e.g., CF mouse/rat, rabbit, ferret, pig) have emerged to optimize the experimental and clinical phases of therapy development including; CFTR modulators, ENAC inhibitors, activation of alternative chloride channels, new antibiotics/anti-inflammatories/mucolytics, phage therapy, cell-based and gene-based therapies. Furthermore, several studies have focused on dissecting the mechanisms of action underlying these therapeutic approaches as well as understanding in depth the impact of mutations on CFTR structure and processing.

This Research Topic aims to gather a collection of reviews and original articles that provide novel information regarding the “Emerging Therapeutic Approaches for Cystic Fibrosis” at basic, translational and clinical levels. This will bring new insights and perspectives in current advances in the field as well as provide managing approaches for this devastating disease.

With special thanks to Dr. Venkateshwar Mutyam for his role in the conception and launch of this Research Topic.

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Editors

Miquéias Lopes-Pacheco

School of Medicine, Emory University

Nicoletta Pedemonte

Giannina Gaslini Institute (IRCCS)

Anthony Kicic

Telethon Kids Institute, University of Western Australia

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241,059 Total views

187,603 Article views

43,559 Article downloads

9,897 Topic views

Published In

Frontiers in Pharmacology

Pharmacology of Ion Channels and Channelopathies

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