WS04.05 Alternative Targets for the Treatment of Cystic Fibrosis Basic Defect

Correction of CF lung abnormalities may be obtained by restoring the function of CFTR with mutation-specific treatments. A potentially alternative approach is to modulate the activity of other targets to stimulate CFTR-independent anion secretion or inhibit acidification. Despite the increasing interest towards possible alternative targets such as TMEM16A, SLC26A9, SLC26A4 and ATP12A, their precise function and expression in the airways is largely unknown or controversial, and specific modulators are still lacking.