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The Effect of CFTR Modulators on Airway Infection in Cystic Fibrosis

The advent of Cystic fibrosis transmembrane receptor (CFTR) modulators in 2012 was a critical event in the history of cystic fibrosis (CF) treatment. Unlike traditional therapies that target downstream effects of CFTR dysfunction, CFTR modulators aim to correct the underlying defect at the protein l...

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Autores principales: Harvey, Caitlyn, Weldon, Sinead, Elborn, Stuart, Downey, Damian G., Taggart, Clifford
Formato: Online Artículo Texto
Lenguaje:English
Publicado: MDPI 2022
Materias:
Acceso en línea:https://www.ncbi.nlm.nih.gov/pmc/articles/PMC8998472/
https://www.ncbi.nlm.nih.gov/pubmed/35408875
http://dx.doi.org/10.3390/ijms23073513
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author Harvey, Caitlyn
Weldon, Sinead
Elborn, Stuart
Downey, Damian G.
Taggart, Clifford
author_facet Harvey, Caitlyn
Weldon, Sinead
Elborn, Stuart
Downey, Damian G.
Taggart, Clifford
author_sort Harvey, Caitlyn
collection PubMed
description The advent of Cystic fibrosis transmembrane receptor (CFTR) modulators in 2012 was a critical event in the history of cystic fibrosis (CF) treatment. Unlike traditional therapies that target downstream effects of CFTR dysfunction, CFTR modulators aim to correct the underlying defect at the protein level. These genotype-specific therapies are now available for an increasing number of CF patients, transforming the way we view the condition from a life-limiting disease to one that can be effectively managed. Several studies have demonstrated the vast improvement CFTR modulators have on normalization of sweat chloride, CFTR function, clinical endpoints, and frequency of pulmonary exacerbation. However, their impact on other aspects of the disease, such as pathogenic burden and airway infection, remain under explored. Frequent airway infections as a result of increased susceptibility and impaired innate immune response are a serious problem within CF, often leading to accelerated decline in lung function and disease progression. Current evidence suggests that CFTR modulators are unable to eradicate pathogenic organisms in those with already established lung disease. However, this may not be the case for those with relatively low levels of disease progression and conserved microbial diversity, such as young patients. Furthermore, it remains unknown whether the restorative effects exerted by CFTR modulators extend to immune cells, such as phagocytes, which have the potential to modulate the response of people with CF (pwCF) to infection. Throughout this review, we look at the potential impact of CFTR modulators on airway infection in CF and their ability to shape impaired pulmonary defences to pathogens.
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spelling pubmed-89984722022-04-12 The Effect of CFTR Modulators on Airway Infection in Cystic Fibrosis Harvey, Caitlyn Weldon, Sinead Elborn, Stuart Downey, Damian G. Taggart, Clifford Int J Mol Sci Review The advent of Cystic fibrosis transmembrane receptor (CFTR) modulators in 2012 was a critical event in the history of cystic fibrosis (CF) treatment. Unlike traditional therapies that target downstream effects of CFTR dysfunction, CFTR modulators aim to correct the underlying defect at the protein level. These genotype-specific therapies are now available for an increasing number of CF patients, transforming the way we view the condition from a life-limiting disease to one that can be effectively managed. Several studies have demonstrated the vast improvement CFTR modulators have on normalization of sweat chloride, CFTR function, clinical endpoints, and frequency of pulmonary exacerbation. However, their impact on other aspects of the disease, such as pathogenic burden and airway infection, remain under explored. Frequent airway infections as a result of increased susceptibility and impaired innate immune response are a serious problem within CF, often leading to accelerated decline in lung function and disease progression. Current evidence suggests that CFTR modulators are unable to eradicate pathogenic organisms in those with already established lung disease. However, this may not be the case for those with relatively low levels of disease progression and conserved microbial diversity, such as young patients. Furthermore, it remains unknown whether the restorative effects exerted by CFTR modulators extend to immune cells, such as phagocytes, which have the potential to modulate the response of people with CF (pwCF) to infection. Throughout this review, we look at the potential impact of CFTR modulators on airway infection in CF and their ability to shape impaired pulmonary defences to pathogens. MDPI 2022-03-23 /pmc/articles/PMC8998472/ /pubmed/35408875 http://dx.doi.org/10.3390/ijms23073513 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
Harvey, Caitlyn
Weldon, Sinead
Elborn, Stuart
Downey, Damian G.
Taggart, Clifford
The Effect of CFTR Modulators on Airway Infection in Cystic Fibrosis
title The Effect of CFTR Modulators on Airway Infection in Cystic Fibrosis
title_full The Effect of CFTR Modulators on Airway Infection in Cystic Fibrosis
title_fullStr The Effect of CFTR Modulators on Airway Infection in Cystic Fibrosis
title_full_unstemmed The Effect of CFTR Modulators on Airway Infection in Cystic Fibrosis
title_short The Effect of CFTR Modulators on Airway Infection in Cystic Fibrosis
title_sort effect of cftr modulators on airway infection in cystic fibrosis
topic Review
url https://www.ncbi.nlm.nih.gov/pmc/articles/PMC8998472/
https://www.ncbi.nlm.nih.gov/pubmed/35408875
http://dx.doi.org/10.3390/ijms23073513
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