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28th Annual North American Cystic Fibrosis Conference

S10: NT: Fruit of the Pipeline: The Latest Clinical Trial Data

Oct 10, 2014 10:30am ‐ Oct 10, 2014 12:20pm

Standard: $49.99


This session will address recent clinical trials that test novel approaches to CF therapies directed at overcoming the basic defect of missing or dysfunctional CFTR.

Learning Objectives:
  • Describe approaches, utility and potential barriers to replacing a defective CFTR using gene therapy.
  • Discuss approaches to correcting F508del CFTR and its potential clinical utility.
  • Describe different potential cellular targets & mechanisms for F508del-directly therapies.


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