[EN] SMALL MOLECULE CFTR CORRECTORS<br/>[FR] CORRECTEURS DE CFTR À PETITES MOLÉCULES
申请人:DISCOVERYBIOMED INC
公开号:WO2014081820A1
公开(公告)日:2014-05-30
Novel CFTR corrector compounds that are effective in rescuing halide efflux, delF508-CFTR protein processing, and apical functional chloride ion transport in a cell are provided. Also provided are methods for treating protein folding disorders (e.g., cystic fibrosis). The methods include administering a CFTR corrector compound or pharmaceutically acceptable salt or prodrug thereof. Methods of rescuing halide efflux in a cell, correcting a processing defect of a delF508-CFTR protein in a cell, and correcting functional delF508-CFTR chloride channels in a cell are also provided.
提供了一种能有效拯救卤化物外流、delF508-CFTR蛋白加工以及细胞顶端功能氯离子输运的新型CFTR校正化合物。还提供了治疗蛋白质折叠障碍(例如囊性纤维化)的方法。这些方法包括给予CFTR校正化合物或其药用盐或前药。还提供了在细胞中拯救卤化物外流、纠正细胞中delF508-CFTR蛋白的加工缺陷以及纠正细胞中功能性delF508-CFTR氯通道的方法。