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Livestreamed Session
Livestreamed Session

PTAC/CFTR
Session Type: Workshop

W11--Topical Advancements in CFTR Biology

Thursday, October 23, 2025
10:15 AM - 12:15 PM PDT
Location:420-422

This session will cover recent discoveries in fundamental mechanisms related to CF biology and physiology. Topics will include CFTR mRNA and protein synthesis, stability, and channel gating, as well as ways in which this knowledge can inform the development of novel therapeutics. In particular, opportunities will be provided to discuss how better understanding CFTR biogenesis is highly relevant to genetic-based therapies.

Learning Objectives:

  • Discuss novel insights into mechanisms of CFTR synthesis and co-translational folding
  • Examine the role of small molecules during modulation of CFTR function
  • Identify improved strategies for delivering and evaluating efficacy of genetic-based therapies

Presentations:

  • 10:15 AM - 12:15 PM PDT
    W11.1- Nonviral delivery of chemically modified suppressor tRNA for overcoming nonsense mutations in cystic fibrosis

    Speaker: Jingan Chen – University of Toronto

  • 10:15 AM - 12:15 PM PDT
    W11.2- Nascent chain misfolding slows translation and stimulates co-translational mRNA decay

    Speaker: Suhee Chang, PhD – Johns Hopkins University School of Medicine

  • 10:15 AM - 12:15 PM PDT
    W11.3- Role of cholesterol on CFTR single channel characteristics and potential cholesterol binding domains

    Speaker: Jonica L. Brown – Emory University

  • 10:15 AM - 12:15 PM PDT
    W11.4- Are there liabilities of codon optimization: Assessment of codon optimization methods in primary Human Bronchial Epithelial (hBE) cells.

    Speaker: Normand Allaire, MS – CFFT Lab, Cystic Fibrosis Foundation, Lexington, MA, USA

  • 10:15 AM - 12:15 PM PDT
    W11.5- Airway targets for CF gene therapies: Development of a tool for the in vitro assessment of anion transport in common and rare cell types of human airway epithelium

    Speaker: Deborah L. Baines, PhD – City St George's, University of London