Cystic fibrosis transmembrane regulator potentiators as promising cystic fibrosis therapies.

摘要

INTRODUCTION: Several types of mutations in the cystic fibrosis transmembrane regulator (CFTR) gene lead to abnormal CFTR protein and alterations of chloride and sodium transmembrane transportation in cystic fibrosis (CF). Some investigational compounds such as VX-770 can improve CFTR protein function. AREAS COVERED: This paper discusses the results of a Phase II study investigating the safety and efficacy of VX-770 in patients with CF. EXPERT OPINION: VX-770 is able to improve chloride and sodium transportation and has a good safety profile. Although such compounds have limited therapeutic targeting potential, preliminary results show great promise in the context of CF therapy.