FIG. 2.

(A) Performance of vectors based on pH221-1-351 and pH221-352-736 in vivo in muscle. Vectors with the human factor IX gene made from AAV1 helpers pH21, pH221-1-351, and pH221-352-736 and AAV2 pH22 were administered to immunodeficient CD4-knockout mice at 10¹¹ particles per animal intramuscularly. The blood obtained by retro-orbital bleeding at week 4 was assayed for human factor IX (hFIX) secretion in mouse plasma. Shown in the figure is the expression level at week 4. (B) The same vectors as in panel A were used in vitro to infect COS cells at a multiplicity of infection of 5,000. The supernatants were harvested at 24 h postinfection. The expression level of human factor IX (hFIX) was measured by ELISA.