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The complex of phosphorylated human cystic fibrosis transmembrane conductance regulator (CFTR) with ATP/Mg and Lumacaftor (VX-809)

ABSTRACT:

SUBMITTER: Jue Chen

PROVIDER: EMPIAR-11008 | biostudies-other |

REPOSITORIES: biostudies-other

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Mechanism of CFTR correction by type I folding correctors.

Fiedorczuk Karol K Chen Jue J

Cell 20220101 1

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 structures of CFTR in complex with the FDA-approved correctors lumacaftor or tezacaftor. ...[more]

PMID: 34995514

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