Fig. 2: Detection of hFVIII-SQ transcript in adult liver biopsy samples. | Nature Medicine

Fig. 2: Detection of hFVIII-SQ transcript in adult liver biopsy samples.

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

Fig. 2

a, Levels of hFVIII-SQ RNA detected in adult liver samples normalized to endogenous reference RNA (YWHAZ). b, Ratio of hFVIII-SQ RNA to circular full-length DNA (R1–R11-linked DNA, PS-DNase + KpnI). c, Representative images of liver biopsy sections from participants who received a dose of 4 × 103 vg per kg body weight (participants 11 and 15; evaluation of one section each), showing hepatocytes that stained positive for hFVIII-SQ RNA by RISH (arrows indicate cytoplasmic staining of AAV5-FVIII-SQ-derived RNA), and the percentage of hepatocytes staining positive for hFVIII-SQ RNA. Images were captured at 1,600 × 1,200 pixels and output at 300 ppi. Scale bars, 20 µm. d, hFVIII-SQ RNA staining signal per cell in 4 × 103 vg per kg body weight-dosed participants; P = 4.24 × 10−29 for participant 11 (n = 2,212 cells examined in one slide) versus participant 15 (n = 1,397 cells examined in one slide), two-tailed unpaired t-test. YWHAZ, tyrosine 3-monooxygenase/tryptophan 5-monooxygenase activation protein zeta.

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