DNA-PKcs Inhibition Improves Sequential Gene Insertion of the Full-Length CFTR cDNA in Airway Stem Cells
Jacob T. Stack, Rachael E. Rayner, Reza Nouri, Carlos Jose Suarez, Sun Hee Kim, Karen L Kanke, Tatyana A. Vetter, Estelle Cormet‐Boyaka, Sriram Vaidyanathan
This study shows that inhibiting DNA repair pathways with AZD7648 improves gene insertion efficiency in airway stem cells for CFTR correction, but may introduce toxicity.
Molecular Therapy — Nucleic Acids · Sep 17, 2024Peer-reviewedGenetics