Figure 2.

Clinical approaches to genome editing strategies for liver diseases: for in vivo gene targeting, viral particles or nanoparticles containing nucleases with or without donor template are injected directly into the patient. For ex vivo gene targeting, hepatocytes are collected from the patient and are treated with nucleases with or without donor template for in situ gene correction or target gene disruption. Alternatively, hiPSCs derived from the patient can be employed as edited target cells and subsequently differentiated into hepatocytes in vitro. Corrected hepatocytes or hiPSC-derived hepatocyte-like cells are then transplanted back into the patient. ZFNs, Zinc finger nucleases; TALENs, Transcription activator-like effector nucleases; CRISPR/Cas9, clustered regularly interspaced short palindromic repeat/(CRISPR)-associated nucleases 9; rAAV, recombinant adeno associated virus; AdV, adenoviral vector; LNP, lipid nanoparticle; hiPSCs, human induced pluripotent stem cells.