Fig. 1. Experimental timeline using microminipigs to observe targeted gene expression in the liver.

On day 0, serum was collected from pigs before injection of the vector, the NAb titer against the AAV8 capsid was determined, and the animals used were selected based on their NAb titer. The AAV8-HCRhAAT-luciferase vector was then injected intravenously, and 7 days later, luciferin was injected from the peripheral vein; gene expression was assessed using an IVIS system. Upon animal sacrifice, the number of vector genome copies from each tissue was analyzed using quantitative PCR.