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30th Annual North American Cystic Fibrosis Conference

W29: NT: Emerging Strategies for Correcting the Basic CFTR Gene Defect

Oct 29, 2016 10:30am ‐ Oct 29, 2016 11:50am

Standard: $49.99


Note: This was a special poster discussion workshop session and its content was not suitable for recording.

Significant advances in gene delivery and gene editing are meeting major progress in pharmacological correction of specific CFTR mutations. These advances have synergized to allow researchers to develop novel cellular and animal based tools in the study of CFTR. These tools are being developed into creative screens to uncover new methods of restoring CFTR function as well as characterizing the mechanisms of known CFTR modulators, correctors and potentiators. This session will provide a broad overview and discussion of multiple approaches to correcting CFTR gene defects.

Educational Objectives:
  • Evaluate the next generation of CFTR modulators, correctors and potentiators.
  • Discuss the latest advances in CFTR gene editing and gene therapy.
  • Discuss the most recent advances in cellular and animal models for assessing CFTR function and identifying novel therapeutic targets.


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