Article
Mechanism of CFTR correction by type I folding correctors.
Cell - 6 Jan 2022
Fiedorczuk Karol, Chen Jue
Abstract excerpt
Small molecule chaperones have been exploited as therapeutics for the hundreds of diseases caused by protein misfolding. The most successful examples are the CFTR correctors, which transformed cystic fibrosis therapy. These molecules revert folding defects of the ΔF508 mutant and are widely used to treat patients. To investigate the molecular mechanism of their action, we determined cryo-electron microscopy...
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