增强剂
囊性纤维化
纤毛
体内
上皮
细胞
医学
效力
气道
功能(生物学)
体外
病理
生物
免疫学
内科学
细胞生物学
生物化学
生物技术
外科
作者
T. J. Neuberger,Bill Burton,Heather A. Clark,Fredrick Van Goor
出处
期刊:Methods in molecular biology
日期:2011-01-01
卷期号:: 39-54
被引量:115
标识
DOI:10.1007/978-1-61779-117-8_4
摘要
The use of human bronchial epithelial (HBE) cell cultures derived from the bronchi of CF patients offers the opportunity to study the effects of CFTR correctors and potentiators on CFTR function and epithelial cell biology in the native pathological environment. Cultured HBE cells derived from CF patients exhibit many of the morphological and functional characteristics believed to be associated with CF airway disease in vivo, including abnormal ion and fluid transport leading to dehydration of the airway surface and the loss of cilia beating. In addition, they can be generated in sufficient quantities to support routine lab testing of compound potency and efficacy and retain reproducible levels of CFTR function over time. Here we describe the development and validation of the CF HBE pharmacology model and its use to characterize, optimize, and select clinical candidates. It is expected that the pre-clinical testing of CFTR potentiators and correctors using epithelial cell cultures derived from CF patients will help to increase their likelihood of clinical efficacy.
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