Correctors of the basic trafficking defect of the mutant F508del-CFTR that causes cystic fibrosis.

Current Opinion in Chemical Biology(2013)

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摘要
•Many correctors of the trafficking defect of mutant CFTR have been identified.•The targets and mechanism of action of most correctors are unknown.•Pharmacological chaperones and proteostasis modulators are defined.•Some correctors are in clinical trials.
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关键词
cystic fibrosis,basic trafficking defect,del-cftr
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