TMEM16A/ANO1: Current Strategies and Novel Drug Approaches for Cystic Fibrosis

Cystic fibrosis (CF) is the most common of rare hereditary diseases in Caucasians, and it is estimated to affect 75,000 patients globally. CF is a complex disease due to the multiplicity of mutations found in the CF transmembrane conductance regulator (CFTR) gene causing the CFTR protein to become d...

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Autores principales:	Christie Mitri, Himanshu Sharma, Harriet Corvol, Olivier Tabary
Formato:	article
Lenguaje:	EN
Publicado:	MDPI AG 2021
Materias:	cystic fibrosis anoctamin-1 calcium-activated chloride channel CFTR-independent therapy Biology (General) QH301-705.5
Acceso en línea:	https://doaj.org/article/63e306b9919c4de784585f452d0ec962
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spelling	oai:doaj.org-article:63e306b9919c4de784585f452d0ec9622021-11-25T17:08:16ZTMEM16A/ANO1: Current Strategies and Novel Drug Approaches for Cystic Fibrosis10.3390/cells101128672073-4409https://doaj.org/article/63e306b9919c4de784585f452d0ec9622021-10-01T00:00:00Zhttps://www.mdpi.com/2073-4409/10/11/2867https://doaj.org/toc/2073-4409Cystic fibrosis (CF) is the most common of rare hereditary diseases in Caucasians, and it is estimated to affect 75,000 patients globally. CF is a complex disease due to the multiplicity of mutations found in the CF transmembrane conductance regulator (CFTR) gene causing the CFTR protein to become dysfunctional. Correctors and potentiators have demonstrated good clinical outcomes for patients with specific gene mutations; however, there are still patients for whom those treatments are not suitable and require alternative CFTR-independent strategies. Although CFTR is the main chloride channel in the lungs, others could, e.g., anoctamin-1 (ANO1 or TMEM16A), compensate for the deficiency of CFTR. This review summarizes the current knowledge on calcium-activated chloride channel (CaCC) ANO1 and presents ANO1 as an exciting target in CF.Christie MitriHimanshu SharmaHarriet CorvolOlivier TabaryMDPI AGarticlecystic fibrosisanoctamin-1calcium-activated chloride channelCFTR-independent therapyBiology (General)QH301-705.5ENCells, Vol 10, Iss 2867, p 2867 (2021)
institution	DOAJ
collection	DOAJ
language	EN
topic	cystic fibrosis anoctamin-1 calcium-activated chloride channel CFTR-independent therapy Biology (General) QH301-705.5
spellingShingle	cystic fibrosis anoctamin-1 calcium-activated chloride channel CFTR-independent therapy Biology (General) QH301-705.5 Christie Mitri Himanshu Sharma Harriet Corvol Olivier Tabary TMEM16A/ANO1: Current Strategies and Novel Drug Approaches for Cystic Fibrosis
description	Cystic fibrosis (CF) is the most common of rare hereditary diseases in Caucasians, and it is estimated to affect 75,000 patients globally. CF is a complex disease due to the multiplicity of mutations found in the CF transmembrane conductance regulator (CFTR) gene causing the CFTR protein to become dysfunctional. Correctors and potentiators have demonstrated good clinical outcomes for patients with specific gene mutations; however, there are still patients for whom those treatments are not suitable and require alternative CFTR-independent strategies. Although CFTR is the main chloride channel in the lungs, others could, e.g., anoctamin-1 (ANO1 or TMEM16A), compensate for the deficiency of CFTR. This review summarizes the current knowledge on calcium-activated chloride channel (CaCC) ANO1 and presents ANO1 as an exciting target in CF.
format	article
author	Christie Mitri Himanshu Sharma Harriet Corvol Olivier Tabary
author_facet	Christie Mitri Himanshu Sharma Harriet Corvol Olivier Tabary
author_sort	Christie Mitri
title	TMEM16A/ANO1: Current Strategies and Novel Drug Approaches for Cystic Fibrosis
title_short	TMEM16A/ANO1: Current Strategies and Novel Drug Approaches for Cystic Fibrosis
title_full	TMEM16A/ANO1: Current Strategies and Novel Drug Approaches for Cystic Fibrosis
title_fullStr	TMEM16A/ANO1: Current Strategies and Novel Drug Approaches for Cystic Fibrosis
title_full_unstemmed	TMEM16A/ANO1: Current Strategies and Novel Drug Approaches for Cystic Fibrosis
title_sort	tmem16a/ano1: current strategies and novel drug approaches for cystic fibrosis
publisher	MDPI AG
publishDate	2021
url	https://doaj.org/article/63e306b9919c4de784585f452d0ec962
work_keys_str_mv	AT christiemitri tmem16aano1currentstrategiesandnoveldrugapproachesforcysticfibrosis AT himanshusharma tmem16aano1currentstrategiesandnoveldrugapproachesforcysticfibrosis AT harrietcorvol tmem16aano1currentstrategiesandnoveldrugapproachesforcysticfibrosis AT oliviertabary tmem16aano1currentstrategiesandnoveldrugapproachesforcysticfibrosis
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TMEM16A/ANO1: Current Strategies and Novel Drug Approaches for Cystic Fibrosis

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