FIG. 1.

Structure of rAAV/Me4βAhFIXm1 and rAAV/CMVhFIXm1 constructs. rAAV/Me4βAhFIXm1 contains the hFIX minigene under the transcriptional control of four tandem copies of the minimal MCK enhancer (Me4; total size 1.2 kb) and 280-bp β-actin promoter. rAAV/CMVhFIXm1 contains the 620-bp CMV immediate-early gene enhancer-promoter, hFIX minigene, and bGH poly(A) signal, flanked by AAV inverted terminal repeats. The CMV promoter probe (0.62-kb EcoRI-BamHI fragment) used for Southern analysis is shown by a dark line. The internal EcoRI fragment detected by the CMV promoter probe is 1.9 kb (arrow a in Fig. 4 and 5). The line at the bottom represents a 5-kb scale, with each mark representing 1 kb.