Research Topic

Emerging Therapeutic Approaches for Cystic Fibrosis

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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 ...

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.


Keywords: cystic fibrosis, CFTR, mutations, therapy development, therapeutic approches, drugs


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