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28th Annual North American Cystic Fibrosis Conference

W11: CFTR-NT: Overcoming Barriers to Fixing CFTR

Oct 10, 2014 2:00pm ‐ Oct 10, 2014 3:45pm

Standard: $49.99


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.

Learning Objectives:
  • 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


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