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PTAC/CFTR
Workshop

W16--From the Gene Editor's Desk: Rewriting Mutant CFTR Using CRISPR & Other Technologies

Friday, September 27, 2024
7:45 AM – 9:45 AM ET
Location:157 A-C

Recorded Session

    Chair(s)

  • Sriram Vaidyanathan

    Nationwide Children’s Hospital, The Ohio State University, Columbus, OH, 43215

  • Greg Newby

    Johns Hopkins University School of Medicine

Genome editing has the potential to serve as a one-time cure for genetic diseases such as cystic fibrosis. This session will include talks from 5 investigators who are pushing the boundaries of several editing methodologies and delivery questions as they apply to cystic fibrosis editing.

Learning Objectives:

  • Mechanistic details of genome editing approaches with promise in treating genetic disease, including homology-directed repair, superexon insertion, base editing, and prime editing.
  • The challenges, prospects, and recent advances towards the treatment of cystic fibrosis with these tools.
  • An update on approaches to deliver genome editing machinery to cells relevant to the treatment of cystic fibrosis.

Presentations:

  • 7:45 AM – 9:45 AM ET
    W16.1- Internal chemical modifications in ssDNA templates enable improved CFTR gene correction and primary airway cell modeling compared to end-modified ssDNA templates

    Speaker: Karen L. Kanke (she/her/hers) – Nationwide Children's Hospital

  • 7:45 AM – 9:45 AM ET
    W16.2- Virus-like particle mediated Cas9/gRNA delivery with a CFTR super exon rescues CFTR function in differentiated primary human bronchial epithelial cells

    Speaker: Ariel Aspiras, PhD – Cystic Fibrosis Foundation

  • 7:45 AM – 9:45 AM ET
    W16.3- Polymeric Nanoparticles for In Utero Gene Delivery in Non-Human Primates

    Speaker: Alexandra S. Piotrowski-Daspit, PhD (she/her/hers) – University of Michigan

  • 7:45 AM – 9:45 AM ET
    W16.4- Rescuing G542X by Adenine Base Editing: A guide to restore function

    Speaker: Lucia Nicosia, MSc – University College Cork

  • 7:45 AM – 9:45 AM ET
    W16.5- Prime editing enables efficient, precise correction of CFTR F508del and functional rescue in human airway epithelial cells

    Speaker: Colin Hemez (he/him/his) – Broad Institute