Modulators of CFTR. Updates on clinical development and future directions

被引：38

作者：

Bardin, Emmanuelle ^{[1
]}

Pastor, Alexandra ^{[2
,3
]}

Semeraro, Michaela ^{[4
]}

Golec, Anita ^{[1
]}

Hayes, Kate ^{[5
]}

Chevalier, Benoit ^{[1
]}

Berhal, Farouk ^{[2
,3
]}

Prestat, Guillaume ^{[2
,3
]}

Hinzpeter, Alexandre ^{[1
]}

Gravier-Pelletier, Christine ^{[2
,3
]}

Pranke, Iwona ^{[1
]}

Sermet-Gaudelus, Isabelle ^{[1
,3
,5
,6
,7
]}

机构：

[1] Inst Necker Enfants Malad, INSERM U1151, Paris, France

[2] UMR CNRS 8601, LCBPT, Paris, France

[3] Univ Paris, Paris, France

[4] Univ Paris, Hop Necker Enfants Malad, Unite Rech Clin CIC P1419, Ctr Invest Clin, Paris, France

[5] European Cyst Fibrosis Soc, Clin Trial Network, Belfast, North Ireland

[6] Hop Necker Enfants Malad, Ctr Reference Malad Rares Mucoviscidose & Malad C, Paris, France

[7] European Resp Network Lung, Paris, France

来源：

EUROPEAN JOURNAL OF MEDICINAL CHEMISTRY | 2021年 / 213卷

关键词：

Cystic fibrosis; CFTR modulators; Corrector; Potentiator; Ivacaftor; Lumacaftor; Tezacafor; Elexacaftor; TRANSMEMBRANE CONDUCTANCE REGULATOR; AGED; 6-11; YEARS; CYSTIC-FIBROSIS; TEZACAFTOR-IVACAFTOR; LUMACAFTOR-IVACAFTOR; FUNCTIONAL SYNERGY; MUTATION; F508DEL-CFTR; POTENTIATOR; CORRECTOR;

D O I：

10.1016/j.ejmech.2021.113195

中图分类号：

R914 [药物化学];

学科分类号：

100701 ;

摘要：

Cystic fibrosis (CF) is the most frequent life-limiting autosomal recessive disorder in the Caucasian population. It is due to mutations in the Cystic Fibrosis Transmembrane Conductance Regulator (CFTR) gene. Current symptomatic CF therapies, which treat the downstream consequences of CFTR mutations, have increased survival. Better knowledge of the CFTR protein has enabled pharmacologic therapy aiming to restore mutated CFTR expression and function. These CFTR "modulators" have revolutionised the CF therapeutic landscape, with the potential to transform prognosis for a considerable number of patients. This review provides a brief summary of their mechanism of action and presents a thorough review of the results obtained from clinical trials of CFTR modulators. (C) 2021 Elsevier Masson SAS. All rights reserved.

引用

页数：15

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