Cystic fibrosis: Insight into CFTR pathophysiology and pharmacotherapy

被引：100

作者：

Lubamba, Bob ^{[1
]}

Dhooghe, Barbara ^{[1
]}

Noel, Sabrina ^{[1
]}

Leal, Teresinha ^{[1
]}

机构：

[1] Catholic Univ Louvain, IREC, Ctr Sci Sante, Louvain Ctr Toxicol & Appl Pharmacol,LTAP, B-1200 Brussels, Belgium

来源：

CLINICAL BIOCHEMISTRY | 2012年 / 45卷 / 15期

关键词：

Cystic fibrosis; CFTR; CFTR pharmacotherapy; CFTR pathophysiology; Chloride transport; TRANSMEMBRANE-CONDUCTANCE-REGULATOR; NASAL POTENTIAL DIFFERENCE; RECEPTOR-MEDIATED ENDOCYTOSIS; EXHALED BREATH CONDENSATE; HUMAN BRONCHIAL EPITHELIA; CHLORIDE CHANNEL; LUNG-DISEASE; PROTEIN-KINASE; STOP MUTATIONS; WILD-TYPE;

D O I：

10.1016/j.clinbiochem.2012.05.034

中图分类号：

R446 [实验室诊断]; R-33 [实验医学、医学实验];

学科分类号：

1001 ;

摘要：

Cystic fibrosis is the most common life-threatening recessively inherited disease in Caucasians. Due to early provision of care in specialized reference centers and more comprehensive care, survival has improved over time. Despite great advances in supportive care and in our understanding of its pathophysiology, there is still no cure for the disease. Therapeutic strategies aimed at rescuing the abnormal protein are either being sought after or under investigation. This review highlights salient insights into pathophysiology and candidate molecules suitable for CFTR pharmacotherapy. Clinical trials using Ataluren, VX-809 and ivacaftor have provided encouraging data. Preclinical data with inhibitors of phosphodiesterase type 5, such as sildenafil and analogs, have highlighted their potential for CFTR pharmacotherapy. Because sildenafil and analogs are in clinical use for other clinical applications, research on this class of drugs might speed up the development of new therapies for CF. (C) 2012 The Canadian Society of Clinical Chemists. Published by Elsevier Inc. All rights reserved.

引用

页码：1132 / 1144

页数：13

共 202 条

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