Correcting mutant CFTR (in particular F508del-CFTR) is a difficult task due to a complex nature of the basic defect. Overcoming the biosynthetic processing arrest, improving defective gating, and increasing the plasma membrane stability are required to fix F508del-CFTR. Several extrinsic factors also limit current rescue strategies. In this session, we will discuss the barriers and present novel approaches to fix mutant CFTR.
Illustrate - To illustrate how the individual's broad genotype may affect the rescue of F508del-CFTR
Explain - To explain how potentiation of CFTR channel activity may modulate protein trafficking and rescue of F508del-CFTR
Discuss - To discuss how external (environmental and microbial) factors reduce the scope of F508del-CFTR correction