Table 1.

Tumorigenesis study

Group/dose	Total studied	Number of mice with tumorsa	Tumors detected
WT	16	3 (19%)	1 (spleen, liver, and lung), 2 (spleen)
KO	30	6 (20%)	2 (lung), 2 (small intestine), 1 (liver), 1 (spleen, kidney, and liver)
2 × 1011 vg/kg	6	2 (33%)	1 (lung), 1 (small intestine and lung)
1012 vg/kg	6	1 (17%)	1 (lung)
2 × 1012 vg/kg	5	2 (40%)	1 (liver), 1 (small intestine and lung)

Specific subsets of animals belonging to different groups were selected at the beginning of the study to investigate the occurrence of tumors at the end of the study (when they were killed at the end of the study, or before the end of the study following the endpoint rule for humanitarian reasons).

^aTumors found in necropsies at the end of the study (age 96 weeks) or after euthanasia.

wt, wild type; KO, knockout; vg, vector genomes.