Genomic Designs of rAAVs Contribute to Pathological Changes in the Livers and Spleens of Mice

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, -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, -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. -6. (e) Endpoint (D194) analysis shows similar pattern to D90 results, hFIX-containing vectors functioned properly throughout the study. -5. ANOVA values reported for both (d) and (e).

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