An overview on chemical structures as ΔF508-CFTR correctors

被引：23

作者：

Spano, Virginia ^{[1
]}

Montalbano, Alessandra ^{[1
]}

Carbone, Anna ^{[1
]}

Scudieri, Paolo ^{[2
]}

Galietta, Luis J. V. ^{[2
,3
]}

Barraja, Paola ^{[1
]}

机构：

[1] Univ Palermo, Chem & Pharmaceut Sci & Technol STEBICEF, Dept Biol, Via Archirafi 32, I-90123 Palermo, Italy

[2] Telethon Inst Genet & Med TIGEM, Campi Flegrei 34, I-80078 Pozzuoli, NA, Italy

[3] Univ Naples Federico II, Dept Translat Med Sci DISMET, I-80131 Naples, Italy

来源：

EUROPEAN JOURNAL OF MEDICINAL CHEMISTRY | 2019年 / 180卷

关键词：

Cystic fibrosis; Cystic fibrosis transmembrane conductance regulator; CFTR; F508del-CFTR; CFTR correctors; TRANSMEMBRANE CONDUCTANCE REGULATOR; SMALL-MOLECULE CORRECTORS; CYSTIC-FIBROSIS; TRAFFICKING DEFECT; QUALITY-CONTROL; PROTEIN; CFTR; CYANOQUINOLINES; IDENTIFICATION; DERIVATIVES;

D O I：

10.1016/j.ejmech.2019.07.037

中图分类号：

R914 [药物化学];

学科分类号：

100701 ;

摘要：

Deletion of phenylalanine at position 508 (F508del) in the CFTR protein, is the most common mutation causing cystic fibrosis (CF). F508del causes misfolding and rapid degradation of CFTR protein a defect that can be targeted with pharmacological agents termed "correctors". Correctors belong to various chemical classes but are generally small molecules based on nitrogen sulfur or oxygen heterocycles. The mechanism of action of correctors is generally unknown but there is experimental evidence that some of them can directly act on mutant CFTR improving folding and stability. Here we overview the characteristics of the various F508del correctors described so far to obtain indications on key chemical structures and modifications that are required for mutant protein rescue. (C) 2019 Elsevier Masson SAS. All rights reserved.

引用

收藏

页码：430 / 448

页数：19

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