CF Amplicons
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 amplicons at on-target and off-target loci from treated cells were sequenced to assess for gene modification, and for off-target genotoxicity. Samples were analyzed by deep sequencing of alleles in the population using Illumina HiSeq sequencer. This study demonstrated site-specific gene editing in cells of interest with low off-target effects.