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…·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.