0       0

33rd Annual North American Cystic Fibrosis Conference


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


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

Description

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.

    Workshop Chair(s):

    Workshop Speaker(s):

    You must be logged in and own this session in order to post comments.

    Print Certificate
    Review Answers
    Print Transcript
    Completed on: token-completed_on
    Review Answers
    Please select the appropriate credit type:
    /
    test_id: 
    credits: 
    completed on: 
    rendered in: 
    * - Indicates answer is required.
    token-content
    token-speaker-name image
    token-speaker-name
    token-index
    token-content
    token-index
    token-content
    token-index
    token-content
    token-index
    token-content
    token-index
    token-content
    token-index
    token-content
    /
    /
    token-index
    token-content