Lung SORT LNPs Enable Precise Homology-Directed Repair Mediated CRISPR/Cas Genome Correction in Cystic Fibrosis Models
This study used Next Generation Sequencing (NGS) to detect gene correction efficiencies in mouse whole lungs after intravenous injections of DOTAP40 lipid nanoparticles encapulating Cas9/sgG542X/HDR template in CF G542X mice.