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33rd Annual North American Cystic Fibrosis Conference

S20--GMS-ET-NT: Molecular Therapies for CF

Nov 2, 2019 2:05pm ‐ Nov 2, 2019 3:50pm


This symposium shows how our understanding of the central dogma of molecular genetics has led to the development of complementary approaches to counteract the effect of CF mutations. Two talks discuss nonsense mutations (PTCs); the first describes novel antisense technology to stabilize CFTR mRNA to enable readthrough, the second introduces a new readthrough approach using engineered tRNAs to suppress PTCs. The third talk describes delivery of chemical modified mRNA directly to the lung in a mouse model of CF which restores CFTR ion channel function and yields improvements in lung function as measured by forced expiratory volume. The final talk focuses on a novel delivery method of CRISPR ribonucleoprotein particles to airway epithelia, a key step in realising the therapeutic potential of gene editing for permanent correction of CF mutations. The approaches described in this session map out how strong basic sciences underpins the therapeutic pipeline for everyone with CF.

Learning Objectives:

  • Define the different classes of mutations that do not respond to currently available modulator therapy.
  • Discuss their cause, and potential correction, in the context of the central dogma of molecular genetics.
  • Apply this understanding to compare and contrast the strengths and weaknesses of the different approaches, and to enable constructive debate on the design and evaluation of future developments of molecular therapies for CF.

Note: Speaker Lulu Huang's presentation was not recorded at her request.

Symposium Chair(s):


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