Article
Repairing the basic defect in cystic fibrosis - one approach is not enough.
The FEBS journal - 1 Jan 2016
Farinha Carlos M, Matos Paulo
Abstract excerpt
Cystic fibrosis has attracted much attention in recent years due to significant advances in the pharmacological targeting of the basic defect underlying this recessive disorder: the deficient functional expression of mutant cystic fibrosis transmembrane conductance regulator (CFTR) chloride channels at the apical membrane of epithelial cells. However, increasing evidence points to the reduced efficacy of single...
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