Cystic fibrosis transmembrane conductance regulator modulators in cystic fibrosis: current perspectives

Béla Z Schmidt,1 Jérémy B Haaf,2 Teresinha Leal,2 Sabrina Noel,2 1Stem Cell Biology and Embryology, Department of Development and Regeneration, Katholieke Universiteit Leuven, Leuven, 2Louvain Center for Toxicology and Applied Pharmacology, Université Cath...

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Autores principales:	Schmidt BZ, Haaf JB, Leal T, Noel S
Formato:	article
Lenguaje:	EN
Publicado:	Dove Medical Press 2016
Materias:	CFTR cystic fibrosis high-throughput screening drug repositioning personalized medicine Therapeutics. Pharmacology RM1-950
Acceso en línea:	https://doaj.org/article/3216efb0df3941889a5dd4ef20f40d88
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spelling	oai:doaj.org-article:3216efb0df3941889a5dd4ef20f40d882021-12-02T01:00:58ZCystic fibrosis transmembrane conductance regulator modulators in cystic fibrosis: current perspectives1179-1438https://doaj.org/article/3216efb0df3941889a5dd4ef20f40d882016-09-01T00:00:00Zhttps://www.dovepress.com/cystic--fibrosis-transmembrane-conductance-regulator-modulators-in-cys-peer-reviewed-article-CPAAhttps://doaj.org/toc/1179-1438Béla Z Schmidt,1 Jérémy B Haaf,2 Teresinha Leal,2 Sabrina Noel,2 1Stem Cell Biology and Embryology, Department of Development and Regeneration, Katholieke Universiteit Leuven, Leuven, 2Louvain Center for Toxicology and Applied Pharmacology, Université Catholique de Louvain, Brussels, Belgium Abstract: Mutations of the CFTR gene cause cystic fibrosis (CF), the most common recessive monogenic disease worldwide. These mutations alter the synthesis, processing, function, or half-life of CFTR, the main chloride channel expressed in the apical membrane of epithelial cells in the airway, intestine, pancreas, and reproductive tract. Lung disease is the most critical manifestation of CF. It is characterized by airway obstruction, infection, and inflammation that lead to fatal tissue destruction. In spite of great advances in early and multidisciplinary medical care, and in our understanding of the pathophysiology, CF is still considerably reducing the life expectancy of patients. This review highlights the current development in pharmacological modulators of CFTR, which aim at rescuing the expression and/or function of mutated CFTR. While only Kalydeco® and Orkambi® are currently available to patients, many other families of CFTR modulators are undergoing preclinical and clinical investigations. Drug repositioning and personalized medicine are particularly detailed in this review as they represent the most promising strategies for restoring CFTR function in CF. Keywords: high-throughput screening, drug repositioning, personalized medicine, precision medicine, potentiators, correctorsSchmidt BZHaaf JBLeal TNoel SDove Medical PressarticleCFTRcystic fibrosishigh-throughput screeningdrug repositioningpersonalized medicineTherapeutics. PharmacologyRM1-950ENClinical Pharmacology: Advances and Applications, Vol Volume 8, Pp 127-140 (2016)
institution	DOAJ
collection	DOAJ
language	EN
topic	CFTR cystic fibrosis high-throughput screening drug repositioning personalized medicine Therapeutics. Pharmacology RM1-950
spellingShingle	CFTR cystic fibrosis high-throughput screening drug repositioning personalized medicine Therapeutics. Pharmacology RM1-950 Schmidt BZ Haaf JB Leal T Noel S Cystic fibrosis transmembrane conductance regulator modulators in cystic fibrosis: current perspectives
description	Béla Z Schmidt,1 Jérémy B Haaf,2 Teresinha Leal,2 Sabrina Noel,2 1Stem Cell Biology and Embryology, Department of Development and Regeneration, Katholieke Universiteit Leuven, Leuven, 2Louvain Center for Toxicology and Applied Pharmacology, Université Catholique de Louvain, Brussels, Belgium Abstract: Mutations of the CFTR gene cause cystic fibrosis (CF), the most common recessive monogenic disease worldwide. These mutations alter the synthesis, processing, function, or half-life of CFTR, the main chloride channel expressed in the apical membrane of epithelial cells in the airway, intestine, pancreas, and reproductive tract. Lung disease is the most critical manifestation of CF. It is characterized by airway obstruction, infection, and inflammation that lead to fatal tissue destruction. In spite of great advances in early and multidisciplinary medical care, and in our understanding of the pathophysiology, CF is still considerably reducing the life expectancy of patients. This review highlights the current development in pharmacological modulators of CFTR, which aim at rescuing the expression and/or function of mutated CFTR. While only Kalydeco® and Orkambi® are currently available to patients, many other families of CFTR modulators are undergoing preclinical and clinical investigations. Drug repositioning and personalized medicine are particularly detailed in this review as they represent the most promising strategies for restoring CFTR function in CF. Keywords: high-throughput screening, drug repositioning, personalized medicine, precision medicine, potentiators, correctors
format	article
author	Schmidt BZ Haaf JB Leal T Noel S
author_facet	Schmidt BZ Haaf JB Leal T Noel S
author_sort	Schmidt BZ
title	Cystic fibrosis transmembrane conductance regulator modulators in cystic fibrosis: current perspectives
title_short	Cystic fibrosis transmembrane conductance regulator modulators in cystic fibrosis: current perspectives
title_full	Cystic fibrosis transmembrane conductance regulator modulators in cystic fibrosis: current perspectives
title_fullStr	Cystic fibrosis transmembrane conductance regulator modulators in cystic fibrosis: current perspectives
title_full_unstemmed	Cystic fibrosis transmembrane conductance regulator modulators in cystic fibrosis: current perspectives
title_sort	cystic fibrosis transmembrane conductance regulator modulators in cystic fibrosis: current perspectives
publisher	Dove Medical Press
publishDate	2016
url	https://doaj.org/article/3216efb0df3941889a5dd4ef20f40d88
work_keys_str_mv	AT schmidtbz cysticfibrosistransmembraneconductanceregulatormodulatorsincysticfibrosiscurrentperspectives AT haafjb cysticfibrosistransmembraneconductanceregulatormodulatorsincysticfibrosiscurrentperspectives AT lealt cysticfibrosistransmembraneconductanceregulatormodulatorsincysticfibrosiscurrentperspectives AT noels cysticfibrosistransmembraneconductanceregulatormodulatorsincysticfibrosiscurrentperspectives
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Cystic fibrosis transmembrane conductance regulator modulators in cystic fibrosis: current perspectives

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