The complex of dephosphorylated human cystic fibrosis transmembrane conductance regulator (CFTR) and Lumacaftor (VX-809)
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ABSTRACT:
SUBMITTER: Jue Chen
PROVIDER: EMPIAR-11006 | biostudies-other |
REPOSITORIES: biostudies-other
Publications
Mechanism of CFTR correction by type I folding correctors.
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]
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2006-03-24 | GSE4513 | GEO
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