Cystic fibrosis (CF) bronchial epithelial cells were treated with nanoparticles containing peptide nucleic acid (PNA) and donor DNA designed to coordinate correction of the F508del CF mutation. PCR am
Various editors were co-expressed in HEK293T cells with the corresponding gRNAs (30/22 for CIRTS and bt1-RESCUE-S,30/26 for ceRBE and S32L14 for CURE, respectively) before on-target editing at the NRA