University of Hertfordshire

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The therapeutic potential of small-molecule modulators of the cystic fibrosis transmembrane conductance regulator (CFTR) Cl- channel. / Liu, Jia; Cami-Kobeci, Gerta; Wang, Yiting; Khuituan, Pissared; Cai, Zhiwei; Li, Hongyu; Husbands, Stephen M.; Sheppard, David N.

Ion Channel Drug Discovery. ed. / Brian Cox; Martin Gosling. Vol. 2015-January 39. ed. Cambridge : Royal Society of Chemistry, 2014. p. 156-185 (RSC Drug Discovery Series).

Research output: Chapter in Book/Report/Conference proceedingChapter

Harvard

Liu, J, Cami-Kobeci, G, Wang, Y, Khuituan, P, Cai, Z, Li, H, Husbands, SM & Sheppard, DN 2014, The therapeutic potential of small-molecule modulators of the cystic fibrosis transmembrane conductance regulator (CFTR) Cl- channel. in B Cox & M Gosling (eds), Ion Channel Drug Discovery. 39 edn, vol. 2015-January, RSC Drug Discovery Series, Royal Society of Chemistry, Cambridge, pp. 156-185. https://doi.org/10.1039/9781849735087-00156

APA

Liu, J., Cami-Kobeci, G., Wang, Y., Khuituan, P., Cai, Z., Li, H., Husbands, S. M., & Sheppard, D. N. (2014). The therapeutic potential of small-molecule modulators of the cystic fibrosis transmembrane conductance regulator (CFTR) Cl- channel. In B. Cox, & M. Gosling (Eds.), Ion Channel Drug Discovery (39 ed., Vol. 2015-January, pp. 156-185). (RSC Drug Discovery Series). Royal Society of Chemistry. https://doi.org/10.1039/9781849735087-00156

Vancouver

Liu J, Cami-Kobeci G, Wang Y, Khuituan P, Cai Z, Li H et al. The therapeutic potential of small-molecule modulators of the cystic fibrosis transmembrane conductance regulator (CFTR) Cl- channel. In Cox B, Gosling M, editors, Ion Channel Drug Discovery. 39 ed. Vol. 2015-January. Cambridge: Royal Society of Chemistry. 2014. p. 156-185. (RSC Drug Discovery Series). https://doi.org/10.1039/9781849735087-00156

Author

Liu, Jia ; Cami-Kobeci, Gerta ; Wang, Yiting ; Khuituan, Pissared ; Cai, Zhiwei ; Li, Hongyu ; Husbands, Stephen M. ; Sheppard, David N. / The therapeutic potential of small-molecule modulators of the cystic fibrosis transmembrane conductance regulator (CFTR) Cl- channel. Ion Channel Drug Discovery. editor / Brian Cox ; Martin Gosling. Vol. 2015-January 39. ed. Cambridge : Royal Society of Chemistry, 2014. pp. 156-185 (RSC Drug Discovery Series).

Bibtex

@inbook{4cbb21c18bd74828b2d055dd1f63fe01,
title = "The therapeutic potential of small-molecule modulators of the cystic fibrosis transmembrane conductance regulator (CFTR) Cl- channel",
abstract = "The cystic fibrosis transmembrane conductance regulator (CFTR) plays a pivotal role in fluid and electrolyte movements across ducts and tubes lined by epithelia. Loss of CFTR function causes the common life-limiting genetic disease cystic fibrosis (CF) and a spectrum of disorders termed CFTR-related diseases, while unphysiological CFTR activity characterises secretory diarrhoea and autosomal dominant polycystic kidney disease (ADPKD). The prevalence of these disorders argues persuasively that small-molecule CFTR modulators have significant therapeutic potential. Here, we discuss how knowledge and understanding of the CFTR Cl− channel, its physiological role and malfunction in disease led to the development of the CFTR potentiator ivacaftor, the first small molecule targeting CFTR approved as a treatment for CF. We consider the prospects for developing other therapeutics targeting directly CFTR including CFTR correctors to rescue the apical membrane expression of CF mutants, CFTR corrector-potentiators, dual-acting small-molecules to correct the processing and gating defects of F508del-CFTR, the commonest CF mutant and CFTR inhibitors to prevent fluid and electrolyte loss in secretory diarrhoea and cyst swelling in ADPKD. The success of ivacaftor provides impetus to other CFTR drug development programmes and a paradigm for the creation of therapeutics targeting the root cause of other genetic disorders",
author = "Jia Liu and Gerta Cami-Kobeci and Yiting Wang and Pissared Khuituan and Zhiwei Cai and Hongyu Li and Husbands, {Stephen M.} and Sheppard, {David N.}",
note = "{\textcopyright} Royal Society of Chemistry 2014",
year = "2014",
month = sep,
day = "18",
doi = "10.1039/9781849735087-00156",
language = "English",
isbn = "978-1-84973-186-7",
volume = "2015-January",
series = "RSC Drug Discovery Series",
publisher = "Royal Society of Chemistry",
pages = "156--185",
editor = "Cox, {Brian } and Gosling, {Martin }",
booktitle = "Ion Channel Drug Discovery",
address = "United Kingdom",
edition = "39",

}

RIS

TY - CHAP

T1 - The therapeutic potential of small-molecule modulators of the cystic fibrosis transmembrane conductance regulator (CFTR) Cl- channel

AU - Liu, Jia

AU - Cami-Kobeci, Gerta

AU - Wang, Yiting

AU - Khuituan, Pissared

AU - Cai, Zhiwei

AU - Li, Hongyu

AU - Husbands, Stephen M.

AU - Sheppard, David N.

N1 - © Royal Society of Chemistry 2014

PY - 2014/9/18

Y1 - 2014/9/18

N2 - The cystic fibrosis transmembrane conductance regulator (CFTR) plays a pivotal role in fluid and electrolyte movements across ducts and tubes lined by epithelia. Loss of CFTR function causes the common life-limiting genetic disease cystic fibrosis (CF) and a spectrum of disorders termed CFTR-related diseases, while unphysiological CFTR activity characterises secretory diarrhoea and autosomal dominant polycystic kidney disease (ADPKD). The prevalence of these disorders argues persuasively that small-molecule CFTR modulators have significant therapeutic potential. Here, we discuss how knowledge and understanding of the CFTR Cl− channel, its physiological role and malfunction in disease led to the development of the CFTR potentiator ivacaftor, the first small molecule targeting CFTR approved as a treatment for CF. We consider the prospects for developing other therapeutics targeting directly CFTR including CFTR correctors to rescue the apical membrane expression of CF mutants, CFTR corrector-potentiators, dual-acting small-molecules to correct the processing and gating defects of F508del-CFTR, the commonest CF mutant and CFTR inhibitors to prevent fluid and electrolyte loss in secretory diarrhoea and cyst swelling in ADPKD. The success of ivacaftor provides impetus to other CFTR drug development programmes and a paradigm for the creation of therapeutics targeting the root cause of other genetic disorders

AB - The cystic fibrosis transmembrane conductance regulator (CFTR) plays a pivotal role in fluid and electrolyte movements across ducts and tubes lined by epithelia. Loss of CFTR function causes the common life-limiting genetic disease cystic fibrosis (CF) and a spectrum of disorders termed CFTR-related diseases, while unphysiological CFTR activity characterises secretory diarrhoea and autosomal dominant polycystic kidney disease (ADPKD). The prevalence of these disorders argues persuasively that small-molecule CFTR modulators have significant therapeutic potential. Here, we discuss how knowledge and understanding of the CFTR Cl− channel, its physiological role and malfunction in disease led to the development of the CFTR potentiator ivacaftor, the first small molecule targeting CFTR approved as a treatment for CF. We consider the prospects for developing other therapeutics targeting directly CFTR including CFTR correctors to rescue the apical membrane expression of CF mutants, CFTR corrector-potentiators, dual-acting small-molecules to correct the processing and gating defects of F508del-CFTR, the commonest CF mutant and CFTR inhibitors to prevent fluid and electrolyte loss in secretory diarrhoea and cyst swelling in ADPKD. The success of ivacaftor provides impetus to other CFTR drug development programmes and a paradigm for the creation of therapeutics targeting the root cause of other genetic disorders

UR - http://www.scopus.com/inward/record.url?scp=84937410581&partnerID=8YFLogxK

U2 - 10.1039/9781849735087-00156

DO - 10.1039/9781849735087-00156

M3 - Chapter

SN - 978-1-84973-186-7

VL - 2015-January

T3 - RSC Drug Discovery Series

SP - 156

EP - 185

BT - Ion Channel Drug Discovery

A2 - Cox, Brian

A2 - Gosling, Martin

PB - Royal Society of Chemistry

CY - Cambridge

ER -