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28th Annual North American Cystic Fibrosis Conference

S01: CFTR: Potentiator Action: Not an Open & Shut Case

Oct 9, 2014 2:00pm ‐ Oct 9, 2014 3:50pm

Standard: $49.99


This session will review our understanding of potentiator mechanism of action and compare the effects that potentiators, such as ivacaftor (VX-770), have on different mutant CFTR proteins. Conclusions that allow for determination of candidates for current potentiator therapies will be drawn. Differences in mutant and wild-type CFTR thermal sensitivity will be discussed. Finally, the impact of potentiators on CFTR correction will be addressed.

Learning Objectives:
  • Describe effects of potentiators on the function of different mutant CFTR proteins.
  • Evaluate effects of potentiators on mutant CFTR thermal stability.
  • Discuss effects of potentiators on correction of mutant CFTR.


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