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APPTT
Workshop

W02--Agnostic Avenues: Alternatives to CF Gene Therapy

Thursday, September 26, 2024
7:45 AM – 9:45 AM ET
Location:153 A-C

Recorded Session

This workshop will provide insights into alternatives to gene therapy. The topics to be covered may include: the effectiveness of synthetic chloride channels in mitigating CF lung disease, strategies for improving CFTR rescue by modulators, modulation of ion channels beyond CFTR, and directly targeting pathophysiological manifestations of CF such as defective mucociliary clearance, infection, and inflammation. The workshop will examine how these strategies can enhance CF lung function and discuss the mechanisms that require further investigation.

Learning Objectives:

  • Expand our knowledge on alternative routes to overcome defective CFTR function in CF airways.
  • Explore strategies to enhance both ion/fluid secretion and absorption to facilitate mucus clearance.
  • Discuss CFTR-independent and mutation-agnostic strategies for enhancing mucus clearance, reducing airway inflammation, and combating infections in people with CF.

Presentations:

  • 7:45 AM – 9:45 AM ET
    W02.1- Anion-selective molecular prosthetics for CFTR outperform amphotericin B in cultured CF epithelia

    Presenting Author: Daniel Greenan, BS (he/him/his) – University of Illinois Urbana-Champaign

  • 7:45 AM – 9:45 AM ET
    W02.2- Primary mouse tracheal basal cells transplanted into CFTR-/- mice reconstitute CFTR function.

    Speaker: Kevin Chen, MA – Center for Regenerative Medicine (CReM) / Boston University School of Medicine

  • 7:45 AM – 9:45 AM ET
    W02.3- FOXI1 deletion alters ion transport and cellular composition in an iPSC-based airway system

    Speaker: Stuart D. Rollins, MD (he/him/his) – Boston Children's Hospital

  • 7:45 AM – 9:45 AM ET
    W02.4- Inhibition of Orai1 with ELD607 is immunomodulatory and reduces chronic lung disease in CF ferrets

    Speaker: Maria Sassano, PhD – Eldec Pharmaceuticals

  • 7:45 AM – 9:45 AM ET
    W02.5- Mechanistic insights into synergistic mucociliary clearance by combined adrenergic and cholinergic agonists

    Speaker: Nam Soo Joo – Stanford University