Figure 2.

Detection of canine factor IX in the mouse liver by immunofluorescence staining. AAV-LSP-cFIX (2.8 × 10¹¹ particles in 200 μl) was injected intraportally into hemophilic C56BL/6 mice. Four months after the injection, one mouse was killed, and the liver was analyzed for canine factor IX by immunofluorescence staining. Plasma level of canine factor IX was 6.5 μg/ml at the time the mouse was killed. Shown is AAV-transduced liver demonstrating the secreted canine factor IX in the linings of sinusoids and central vein (a, ×20; b, ×40). (c) Control naive mouse (×40). (d–f) Transduction of mouse liver by AAV-LSP-GFP. Particles (3 × 10¹¹) in 150 μl were injected intraportally into C56BL/6 mice. Eighteen weeks after the injection, one mouse was killed, and the liver was analyzed for GFP expression by immunofluorescence microscopy. Hepatocytes positive for GFP expression were identified in the AAV-transduced liver. [d, ×20; e, ×40; f (control naive mouse), ×40].