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Measurements of Functional Responses in Human Primary Lung Cells as a Basis for Personalized Therapy for Cystic Fibrosis

机译:测量人原代肺细胞功能反应作为囊性纤维化个性化治疗的基础

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摘要

BackgroundThe best investigational drug to treat cystic fibrosis (CF) patients with the most common CF-causing mutation (F508del) is VX-809 (lumacaftor) which recently succeeded in Phase III clinical trial in combination with ivacaftor. This corrector rescues F508del-CFTR from its abnormal intracellular localization to the cell surface, a traffic defect shared by all Class II CFTR mutants. Our goal here is to test the efficacy of lumacaftor in other Class II mutants in primary human bronchial epithelial (HBE) cells derived from CF patients.
机译:背景技术治疗具有最常见的CF致突变(F508del)的囊性纤维化(CF)患者的最佳研究药物是VX-809(lumacaftor),该药物最近与ivacaftor结合在III期临床试验中获得了成功。该校正子将F508del-CFTR从其异常的细胞内定位拯救到细胞表面,这是所有II类CFTR突变体共有的交通缺陷。我们的目标是在源自CF患者的原代人支气管上皮(HBE)细胞中测试lumacaftor在其他II类突变体中的功效。

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