Fig. 5: LV biodistribution in treated NHP. | Nature Communications

Fig. 5: LV biodistribution in treated NHP.

From: Liver-directed lentiviral gene therapy corrects hemophilia A mice and achieves normal-range factor VIII activity in non-human primates

Fig. 5: LV biodistribution in treated NHP.

a Single values of VCN in the indicated organs of LV-treated NHP, as indicated, at necropsy (60 days post-LV). The dashed lines defining the gray area represent the lower limit of detection (0.0003) and the lower limit of reliable quantification (0.007, see methods section). b Single values and mean with SEM of expression analysis by quantitative PCR of WPRE normalized on the endogenous TAF7 gene on RNA extracted from five different liver lobes of LV-treated NHP, as indicated. The gray area represents mean background signal for WPRE expression in the spleen. c Single values of VCN measured or calculated (*) in the indicated liver subpopulation after gradient purification (nPC) or FACS-sorting (KC, Lsec). VCN in Hep is calculated according to the formula VCNliver = (VCNHep × 0.7) + (VCNnPC × 0.3). This formula is based on average proportion of liver cell subpopulations in the liver.

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