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33rd Annual North American Cystic Fibrosis Conference

W25--ET-NT: Addressing Unmet Needs for Nonsense & Rare Mutations

Nov 2, 2019 10:30am ‐ Nov 2, 2019 11:50am


This workshop highlights advancements in new and emerging technologies progressing towards a cure. Discussions will include development of tools/strategies focused on rare and nonsense CFTR variants with translatable relevance to all CFTR mutations.

Learning Objectives:

  • Summarize molecular defects attributable to CFTR premature truncation codons, including the role of nonsense mediated decay and its potential as a therapeutic target.
  • Compare approaches for CFTR gene repair or replacement in primary airway epithelia.
  • Evaluate state-of-the-art directed differentiation of airway progenitor cells from pluripotent source material.

Note: Speaker Youngjin Kim's presentation was not recorded at his request.

Workshop Chair(s):

Workshop Speaker(s):

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