EPS3.04 Rescuing rare CFTR mutants by a mimetic peptide targeting the AKAP function of PI3Kγ

Abstract

The approval of CFTR modulators has opened the possibility of targeting the basic molecular defects underlying CF. Nevertheless, these molecules fail to completely rescue the activity of CFTR mutants, and patients with rare mutations are not eligible for these treatments. We previously conceived a cell-permeable mimetic peptide (PI3Kγ MP) that, by targeting PI3Kγ, induces cAMP elevation in CFTR proximity triggering PKA-mediate phosphorylation and CFTR opening.