SCHWIEBERT, ERIK M.,COLLAWN, JAMES F.,BEBOK, ZSUZSA
申请号：
CA2836463
公开号：
CA2836463A1
申请日:
2012.05.17
申请国别(地区):
CA
年份:
2012
代理人:
摘要：
Novel CFTR corrector compounds that are effective in rescuing halide efflux 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 screening for CFTR corrector compounds are also described herein. The methods of screening include contacting a cell that endogenously expresses a CFTR mutation with the compound to be screened and detecting a rescue of halide efflux from the cell.
