Skip to main content
NACFC 2025 Main banner
Icon Legend
This session is not in your schedule.
This session is in your schedule. Click again to remove it.
Presentation Icons
Session includes an in person invitee/speaker with CF
Session includes an in person invitee/speaker with CF
Livestreamed Session
Livestreamed Session
Recorded Session
Recorded Session
Session is Ticketed (Must Register for Session through Registration)
Session is Ticketed (Must Register for Session through Registration)
Food Available for Session Attendees
Food Available for Session Attendees
Session Feedback

PTAC/CFTR
Session Type: Workshop

W30--Gene Editing: Delivery of Gene Editors | Room: Signature Room (Level 5)

Saturday, October 25, 2025
7:45 AM - 9:45 AM PDT
Location:Signature Room (Level 5)

Recorded Session

This session will focus on efforts to effectively deliver genome editors into epithelial cells in vivo to enable highly efficient in vivo gene correction. Speakers will present their work on the use of polymeric and lipid nanoparticles to deliver genome editors. In addition, the session will include talks discussing emerging reporter mice that may help optimize gene delivery in vivo.

Learning Objectives:

  • Update on efforts to optimize delivery of genome editors in vivo using lipid and polymeric nanoparticles to enable efficient gene correction
  • Highlight emerging model systems that may help evaluate efficacy of gene delivery and genome editing in vivo
  • Update on in utero delivery of genome editors

Presentations:

  • 7:45 AM - 9:45 AM PDT
    W30.1- Polymeric nanoparticle-mediated adenine base editing targets key airway epithelial cell types for functional restoration of a common CFTR splice variant

    Speaker: Erin W. Kavanagh, n/a – Johns Hopkins University School of Medicine

  • 7:45 AM - 9:45 AM PDT
    W30.2- A murine CF reporter system to assess delivery and duration of gene editing and cell transplantation therapeutics

    Speaker: Karen K. Schelde, n/a – Case Western Reserve University

  • 7:45 AM - 9:45 AM PDT
    W30.3- Antibody/peptide-nanoparticle conjugates for specific cell-type targeting of genetic therapies

    Speaker: Owen Kelly, B.S. in Biomedical Engineering – University of Michigan

  • 7:45 AM - 9:45 AM PDT
    W30.4- CANCELLED: Optimizing prime editing for precise correction of the CFTR G542X mutation via LNP-mediated delivery

    Speaker: Tyler Thomson, n/a – University of Toronto

  • 7:45 AM - 9:45 AM PDT
    W30.5- Ionizable lipid nanoparticles for gene editing of the fetal lung

    Speaker: Adele Ricciardi, MD, PhD – University of Pennsylvania and Children's Hospital of Philadelphia