W30--Gene Editing: Delivery of Gene Editors

Chair(s)

• 

  Gregory Newby

  Johns Hopkins University
• 

  Sriram Vaidyanathan, PhD

  Pediatrics
  Nationwide Children's Hospital

This section 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.

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 Kavanagh, PhD – Johns Hopkins University School of Medicine
• 7:45 AM - 9:45 AM PDT

  W30.2- A murine Cystic Fibrosis reporter system to assess delivery and duration of gene editing and cell transplantation therapeutics
  

  Speaker: Karen K. Schelde, PhD – 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- 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