发明名称 |
TREATING PROTEIN FOLDING DISORDERS WITH SMALL MOLECULE CFTR CORRECTORS |
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. |
申请公布号 |
US2014073632(A1) |
申请公布日期 |
2014.03.13 |
申请号 |
US201214117705 |
申请日期 |
2012.05.17 |
申请人 |
SCHWIEBERT ERIK M.;COLLAWN JAMES F.;BEBOK ZSUZSA;THE UAB RESEARCH FOUNDATION;DISCOVERYBIOMED INC. |
发明人 |
SCHWIEBERT ERIK M.;COLLAWN JAMES F.;BEBOK ZSUZSA |
分类号 |
C07D513/04;C07D223/04;C07D239/42;C07D241/52;C07D251/46;C07D417/04;C07D473/06;C07D487/04 |
主分类号 |
C07D513/04 |
代理机构 |
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代理人 |
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主权项 |
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地址 |
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