Fig. 2.

Gene editing-based therapy in human albumin locus. 1. Gene editing-based therapy for MPS I or II patients targets albumin locus sites in the liver. 2. ZFN binds to DNA sequences in the albumin locus and their fok1 nuclease domains dimerize between the binding sites. 3. ZFN induces DSB at the albumin locus in the genome of hepatocytes. 4. Alpha-L-iduronidase or iduronate-2-sulfatase genes are inserted into the albumin locus, and their proteins are continually produced from the liver into the blood. Abbreviation: ZFN, zinc finger nucleases; DSB, double-strand breaks