VX-809 and Related Corrector Compounds Exhibit Secondary Activity Stabilizing Active F508del-CFTR after Its Partial Rescue to the Cell Surface
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- 作者：Paul D.W. Eckford1 ; 5 ; Mohabir Ramjeesingh1 ; 5 ; Steven Molinski1 ; Stan Pasyk1 ; Johanna F. Dekkers4 ; Canhui Li1 ; Saumel Ahmadi1 ; Wan Ip2 ; Timothy E. Chung1 ; Kai Du3 ; Herman Yeger3 ; Jeffrey Beekman4 ; Tanja Gonska2 ; Christine E. Bear1 ; bear@sickkids.ca" class="auth_mail
- 刊名：Chemistry & Biology
- 出版年：22 May 2014
- 年：2014
- 卷：21
- 期：5
- 页码：666-678
- 全文大小：2727 K
文摘
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The intrinsic instability of F508del-CFTR is ameliorated by certain correctors
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A corrector binds purified F508del-CFTR in proteoliposomes to enhance activity
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F508del-CFTR is rescued via cotranslational and posttranslational mechanisms
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The mechanism of action of small molecules was studied in patient-derived organoids