Figure 6.

Vector biodistribution and hepatotoxicity. A) VCNs showed wide variation with most subjects expressing 1 vector copy every 10–1000 cells in the absence of specific organ tropism. B) Regardless of initial VCN, all tissues showed a substantial decline in tissue vector load of 1–3-log-fold every 12 mo; there was no difference between AAV5- and AAV8-treated animals. C) Liver-specific ALT levels increased intermittently, particularly in AAV8 recipients, but overall transaminases remained in the normal range.