CFTR Modulators: Shedding Light on Precision Medicine for Cystic Fibrosis.

ABSTRACT: Cystic fibrosis (CF) is the most common life-threatening monogenic disease afflicting Caucasian people. It affects the respiratory, gastrointestinal, glandular and reproductive systems. The major cause of morbidity and mortality in CF is the respiratory disorder caused by a vicious cycle of obstruction of the airways, inflammation and infection that leads to epithelial damage, tissue remodeling and end-stage lung disease. Over the past decades, life expectancy of CF patients has increased due to early diagnosis and improved treatments; however, these patients still present limited quality of life. Many attempts have been made to rescue CF transmembrane conductance regulator (CFTR) expression, function and stability, thereby overcoming the molecular basis of CF. Gene and protein variances cau

SUBMITTER: Lopes-Pacheco M 

PROVIDER: S-EPMC5011145 | biostudies-literature | 2016

REPOSITORIES: biostudies-literature