Efficient in vivo editing of OTC-deficient patient-derived primary human hepatocytes.

•Therapeutically relevant levels of single-nucleotide repair of the human OTC locus were achieved in vivo.•Single-nucleotide editing of primary human hepatocytes was facilitated by a highly hepatotropic bioengineered AAV capsid.•A novel human minigene platform proved highly effective for evaluation of human liver-specific genome editing reagents.