Cystic Fibrosis (CF) is a serious genetic condition caused by CF transmembrane conductance
regulator (CFTR) mutation. CF patients have shortened lifespan due to airway obstruction, infection,
and end-stage lung failure. However, recent development in CF therapy suggests a brighter future
for CF patients. Targeting specific CFTR mutations aims to potentiate the channel gating activity of
impaired CFTR and restore protein trafficking to the plasma membrane. Gene therapy introduces correct
CFTR gene into the affected airway epithelium leading to the functional expression of CFTR in
CF patients. This review will sum up the current status in CF-cause targeting therapy.
Keywords: CFTR, chloride transporter, corrector, cystic fibrosis, gene therapy, potentiator.
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