Restoration of F508-del Function by Transcomplementation: The Partners Meet in the Endoplasmic Reticulum.

Emily Anne Smith Bergbower,Inna Sabirzhanova,Clément Boinot,William B Guggino,Liudmila Cebotaru

Cellular physiology and biochemistry : international journal of experimental cellular physiology, biochemistry, and pharmacology（2019）

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摘要

We conclude that the functional rescue of F508-del by transcomplementation occurs via a bimolecular interaction that most likely begins in the ER and continues at the plasma membrane. These results come at an opportune time for developing a gene therapy for CF and offer new treatment options for a wide range of CF patients.

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关键词

Bimolecular interaction,Cystic fibrosis,Gene therapy,Transcomplementation,Truncated CFTR

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