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CFTR Modulator Therapies: Potential Impact on Airway Infections in Cystic Fibrosis

Cystic Fibrosis (CF) is an autosomal recessive disease caused by mutations in the gene encoding for the Cystic Fibrosis Transmembrane conductance Regulator (CFTR) protein, expressed on the apical surface of epithelial cells. CFTR absence/dysfunction results in ion imbalance and airway surface dehydr...

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Autores principales: Saluzzo, Francesca, Riberi, Luca, Messore, Barbara, Loré, Nicola Ivan, Esposito, Irene, Bignamini, Elisabetta, De Rose, Virginia
Formato: Online Artículo Texto
Lenguaje:English
Publicado: MDPI 2022
Materias:
Acceso en línea:https://www.ncbi.nlm.nih.gov/pmc/articles/PMC8998122/
https://www.ncbi.nlm.nih.gov/pubmed/35406809
http://dx.doi.org/10.3390/cells11071243
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author Saluzzo, Francesca
Riberi, Luca
Messore, Barbara
Loré, Nicola Ivan
Esposito, Irene
Bignamini, Elisabetta
De Rose, Virginia
author_facet Saluzzo, Francesca
Riberi, Luca
Messore, Barbara
Loré, Nicola Ivan
Esposito, Irene
Bignamini, Elisabetta
De Rose, Virginia
author_sort Saluzzo, Francesca
collection PubMed
description Cystic Fibrosis (CF) is an autosomal recessive disease caused by mutations in the gene encoding for the Cystic Fibrosis Transmembrane conductance Regulator (CFTR) protein, expressed on the apical surface of epithelial cells. CFTR absence/dysfunction results in ion imbalance and airway surface dehydration that severely compromise the CF airway microenvironment, increasing infection susceptibility. Recently, novel therapies aimed at correcting the basic CFTR defect have become available, leading to substantial clinical improvement of CF patients. The restoration or increase of CFTR function affects the airway microenvironment, improving local defence mechanisms. CFTR modulator drugs might therefore affect the development of chronic airway infections and/or improve the status of existing infections in CF. Thus far, however, the full extent of these effects of CFTR-modulators, especially in the long-term remains still unknown. This review aims to provide an overview of current evidence on the potential impact of CFTR modulators on airway infections in CF. Their role in affecting CF microbiology, the susceptibility to infections as well as the potential efficacy of their use in preventing/decreasing the development of chronic lung infections and the recurrent acute exacerbations in CF will be critically analysed.
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spelling pubmed-89981222022-04-12 CFTR Modulator Therapies: Potential Impact on Airway Infections in Cystic Fibrosis Saluzzo, Francesca Riberi, Luca Messore, Barbara Loré, Nicola Ivan Esposito, Irene Bignamini, Elisabetta De Rose, Virginia Cells Review Cystic Fibrosis (CF) is an autosomal recessive disease caused by mutations in the gene encoding for the Cystic Fibrosis Transmembrane conductance Regulator (CFTR) protein, expressed on the apical surface of epithelial cells. CFTR absence/dysfunction results in ion imbalance and airway surface dehydration that severely compromise the CF airway microenvironment, increasing infection susceptibility. Recently, novel therapies aimed at correcting the basic CFTR defect have become available, leading to substantial clinical improvement of CF patients. The restoration or increase of CFTR function affects the airway microenvironment, improving local defence mechanisms. CFTR modulator drugs might therefore affect the development of chronic airway infections and/or improve the status of existing infections in CF. Thus far, however, the full extent of these effects of CFTR-modulators, especially in the long-term remains still unknown. This review aims to provide an overview of current evidence on the potential impact of CFTR modulators on airway infections in CF. Their role in affecting CF microbiology, the susceptibility to infections as well as the potential efficacy of their use in preventing/decreasing the development of chronic lung infections and the recurrent acute exacerbations in CF will be critically analysed. MDPI 2022-04-06 /pmc/articles/PMC8998122/ /pubmed/35406809 http://dx.doi.org/10.3390/cells11071243 Text en © 2022 by the authors. https://creativecommons.org/licenses/by/4.0/Licensee MDPI, Basel, Switzerland. This article is an open access article distributed under the terms and conditions of the Creative Commons Attribution (CC BY) license (https://creativecommons.org/licenses/by/4.0/).
spellingShingle Review
Saluzzo, Francesca
Riberi, Luca
Messore, Barbara
Loré, Nicola Ivan
Esposito, Irene
Bignamini, Elisabetta
De Rose, Virginia
CFTR Modulator Therapies: Potential Impact on Airway Infections in Cystic Fibrosis
title CFTR Modulator Therapies: Potential Impact on Airway Infections in Cystic Fibrosis
title_full CFTR Modulator Therapies: Potential Impact on Airway Infections in Cystic Fibrosis
title_fullStr CFTR Modulator Therapies: Potential Impact on Airway Infections in Cystic Fibrosis
title_full_unstemmed CFTR Modulator Therapies: Potential Impact on Airway Infections in Cystic Fibrosis
title_short CFTR Modulator Therapies: Potential Impact on Airway Infections in Cystic Fibrosis
title_sort cftr modulator therapies: potential impact on airway infections in cystic fibrosis
topic Review
url https://www.ncbi.nlm.nih.gov/pmc/articles/PMC8998122/
https://www.ncbi.nlm.nih.gov/pubmed/35406809
http://dx.doi.org/10.3390/cells11071243
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