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AAV-FVIII gene transfer in the presence of FVIII immunity. Copy number and mRNA levels were determined in hepatocytes isolated from the perfused livers of naive hemophilia A mice preimmunized with ET3 and treated with 4 × 1012 vp/kg AAV8-HLP-ET3 vector (n = 4). Dashed lines and shaded regions indicate the limit of detection for determining both transgene and mRNA copy number. AAV, adeno-associated virus; HLP, hybrid liver-specific promoter.
