Extended Data Fig. 2: Valoctocogene roxaparvovec transduction patterns in hepatocytes amongst the four participants with > 2% hepatocytes stained positive. | Nature Medicine

Extended Data Fig. 2: Valoctocogene roxaparvovec transduction patterns in hepatocytes amongst the four participants with > 2% hepatocytes stained positive.

From: Interindividual variability in transgene mRNA and protein production following adeno-associated virus gene therapy for hemophilia A

Extended Data Fig. 2

In situ hybridization analysis to detect vector genomes, showing unbiased distribution of hFVIII-SQ DNA (brown foci) in liver biopsy samples of Participants 11, 15, 3, and 4; Participant 1 has less than 2% of hepatocytes stained positive for vector DNA and did not have any identifiable veins (PC or PV) as the biopsy was broken into many pieces, thus not shown. Each focus (brown dot) represents at least one vector DNA molecule; it is possible to have multiple copies of vector genome within a single focus. Zoom-out (widefield) images allow comparison of liver sections proximal and distal to central veins. Zoom-in images separated according to liver zones – zone 1 being peri-portal vein (PP), zone 3 being peri-central vein (PC), and zone 2 being in between zone 1 and 3. Images were captured at 1600 × 1200 pixels and output at 300 ppi. PV, portal vein.

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