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Fig. 7 | Genome Biology

Fig. 7

From: Curing hemophilia A by NHEJ-mediated ectopic F8 insertion in the mouse

Fig. 7

Long-term efficacy and safety in BDDF8-edited hemophilia A mice. a Long-term follow-up on the F8 activity of hemophilia A mice. The animals were hydrodynamically injected with Cas9-sgAlb and the double-cut donor pD-BDDF8-sg (n = 15). The exact P value is shown by a one-way ANOVA analysis. b Treated mice survive a tail-clip challenge. Wild-type C57BL/6 (WT) mice (n = 5) served as a positive control. c Hematoxylin and eosin (H&E) staining of the liver sections of untreated and treated HA mice 1 year after injection. Shown are representative images from five mice. d Liver toxicity markers 1 year after treatment. AST, aspartate aminotransferase; ALT, alanine aminotransferase; TBIL, total bilirubin; Alb, total albumin (n = 10). No significant differences were observed between untreated HA mice (n = 10) and treated HA mice (n = 10) by unpaired t test with Welch’s correction. e Levels of F8 inhibitors in mouse plasma measured by Bethesda assay. Untreated 1 year (n = 8); treated (n = 8). An unpaired t test with Welch’s correction was used for statistical analysis. ns, not significant. f Two-photon imaging of liver tissues indicates a stable expression of tdTomato. CD144 (VE-cadherin) stains the liver vasculature structure; edited cells (tdTomato-BDDF8) were pseudo-colored as green. Shown is a representative image of n = 4 mice. g ddPCR analysis indicates the long-term presence of junctions of NHEJ mediated knock-in 1 year after treatment. Schematic and detailed information was presented in Fig. 3

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