[HTML][HTML] Correcting CFTR folding defects by small-molecule correctors to cure cystic fibrosis

M Mijnders, B Kleizen, I Braakman - Current opinion in pharmacology, 2017 - Elsevier
Highlights•Combining correctors that correct different folding defects improves
efficacy.•Complete F508del CFTR rescue likely requires full correction of NBD1.•All classes
of CF-causing mutations may benefit from correctors.•F508del CFTR can be rescued from
the ER by proteostasis regulators and amplifiers.•The drug development pipeline contains
promising CFTR corrector combinations.Pharmacological intervention to treat the lethal
genetic disease cystic fibrosis has become reality, even for the severe, most common folding …