Figure 5.

Experimental setup and results of in vivo transductions in xenograft model of the human liver. (A) Schematic representation of the AAV Testing Kit study performed in hFRG animal. (B) Results of AAV vector copy number quantification in selected organs. The values are given as vcn/dg. The quantity of endogenous genome was obtained by measuring the copy number of human genomic albumin locus in genomic DNA purified from human hepatocytes, while murine albumin genomic locus was targeted in mouse hepatocytes and other organs. (C) Results of NGS-based quantification of vector-encoded DNA in selected tissues. Values are given as percentage of total reads for each organ. (D) Results of NGS analysis of vector function based on quantification of vector-encoded transgene mRNA. Values are given as percentage of total reads for each cell type. The values presented in panels (C, D) were rounded to the nearest whole number. i.v., intravenous.