Small molecule CFTR correctors

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专利权人:

DiscoveryBioMed; Inc.

发明人:

Schwiebert, Erik,Streiff, John,Dixon, John,Gao, Hongwu

申请号：

AU2013348018

公开号：

AU2013348018A1

申请日:

2013.11.20

申请国别(地区):

AU

年份:

2015

代理人:

摘要：

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.

来源网站：

中国工程科技知识中心

来源网址：

http://www.ckcest.cn/home/