Article

Mechanism of CFTR correction by type I folding correctors

2021-06-18

Abstract excerpt

<h4>SUMMARY</h4> 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. However, their mechanism of action is unknown. Here we present cryo-electron micros...

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Literature Corpus work: 0f637ad1-bc34-5bbf-997c-162e853b0e09
DOI: 10.1101/2021.06.18.449063

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