Fig. 1: Schematic illustration of SORT LNP-mediated gene correction therapy in CF mouse models.

SORT LNPs encapsulating three nucleic acid cargos (Cas9 mRNA, sgRNA, and ssDNA HDR template) effectively corrected CFTR gene mutations in a G542X CF mouse model (in vivo) and in a patient-derived F508del HBE cell model (ex vivo).