发明名称 |
TREATING PROTEIN FOLDING DISORDERS WITH SMALL MOLECULE CFTR CORRECTORS |
摘要 |
<p>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.</p> |
申请公布号 |
EP2709628(A2) |
申请公布日期 |
2014.03.26 |
申请号 |
EP20120785637 |
申请日期 |
2012.05.17 |
申请人 |
DISCOVERYBIOMED INC.;THE UAB RESEARCH FOUNDATION |
发明人 |
SCHWIEBERT, ERIK M.;COLLAWN, JAMES F.;BEBOK, ZSUZSA |
分类号 |
A61K31/519;A61K31/4164;A61K31/505;A61P11/00 |
主分类号 |
A61K31/519 |
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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