Figure 6:

HemB mice injected with rAAVs containing incomplete genomes exhibit reduced liver: spleen weight ratios and greater clot time compared to TTR-hFIX-injected mice. (a) Liver weight mice trended towards significantly different. Kruskal-Wallis test performed, N = 3–8. (b) Spleen weights reveal some mice with very heavy spleens. TTR-hFIX had lowest average spleen weight of vector-injected groups. Red dots: moribund mice, Kruskal-Wallis test performed, N = 3–5. (c) Liver to spleen weight ratios of HemB mice injected with rAAVs. TTR-hFIX mice and controls are higher than other four groups. CB and CB-PolyA ratios are significantly lower compared to TTR-hFIX. (d) Clot time analysis of HemB mice via APTT at D90. N = 3–6. (e) Endpoint (D194) analysis shows similar pattern to D90 results, hFIX-containing vectors functioned properly throughout the study. N = 2–5. ANOVA p values reported for both (d) and (e).