Fig. 4: VWF expression in VWD mice administered with endothelial or liver-specific dual hybrid AAV vectors. | Gene Therapy

Fig. 4: VWF expression in VWD mice administered with endothelial or liver-specific dual hybrid AAV vectors.

From: Development of a dual hybrid AAV vector for endothelial-targeted expression of von Willebrand factor

Fig. 4: VWF expression in VWD mice administered with endothelial or liver-specific dual hybrid AAV vectors.

a Scheme of the study design. AAV vectors were injected in VWF deficient mice (VWF−/−, n = 5) at a dose of 5 × 1011 vg/mouse. Red symbols represent timing of blood collection. Sac sacrifice. b Graph reporting VWF expression levels in plasma over time, expressed as percentage relative to the average levels of wt mice (set as 100%). c Graph reporting the measurement of FVIII activity (% FVIIIa:C) in plasma samples collected from mice 1 week before and 2 weeks after AAV injection.

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