Lipid Nanoparticles for the Delivery of CRISPR/Cas9 Machinery to Enable Site-Specific Integration of CFTR and Mutation-Agnostic Disease Rescue
Ruth A. Foley, Paul George Ayoub, Vrishti Sinha, Colin J. Juett, Alicia Sanoyca, Emily C. Duggan, Lindsay E. Lathrop, Priyanka Bhatt, Kevin J. Coote, Beate Illek, Brigitte N. Gomperts, Donald B. Kohn, Steven John Jonas
This paper develops lipid nanoparticles to deliver CRISPR/Cas9 for site-specific CFTR gene integration in CF airway cells, achieving 3-3.5% gene editing and 80% Cl- current restoration.
Advanced Functional Materials · Jan 4, 2026Peer-reviewedBioengineering