Table 1. Summary of experimental details.
Specifics are shown for all animals or study groups used for the morphometric analysis and shown in figures 1, 2, 3, and 6. The type of polyadenylation signal (SV40, simian virus 40; bGH, bovine growth hormone) and the presence or absence of a woodchuck hepatitis post-transcriptional regulatory element (+WPRE; -WPRE) are indicated for each vector. Additional details about vectors are included in materials and methods. All animals were male except for dog M2396. The gender of newborn mice was not determined.
| Species | Vector | Dose | Route of injection | Age at injection | Time of analysis after injection |
|---|---|---|---|---|---|
| mice, group 1 (spf-ash) | scAAV8.TBG.hOTCco (SV40/−WPRE) | 1×1010 GC per animal | tail vein | 3-6 months | 28 days |
| mice, group 2 (C57BL/6) | AAV8.TBG.EGFP (bGH/+WPRE) | 1×1011 GC per animal | tail vein | 6-8 weeks | 7 days |
| mice, group 3 (C57BL/6 LDLR-/-APOBEC1-/-) | AAV8.TBG.PI.EGFP-miRscrT.bGH (bGH/+WPRE) | 3×1011 GC per animal | tail vein | 6-8 weeks | 14 days |
| mice, group 4 (C57BL/6) | scAAV8.TBG.EGFP (SV40/−WPRE) | 3×109 GC per animal | tail vein | 6-8 weeks | 7 days |
| newborn mice, group 5 (C57BL/6) | AAV8.TBG.EGFP (bGH/+WPRE) | 2.5×1010 GC per animal | temporal vein | ≤ 24 h | 7 days |
| newborn mice, group 6 (C57BL/6) | scAAV8.TBG.EGFP (SV40/−WPRE) | 5×1010 GC per animal | temporal vein | ≤ 24 h | 4-7 days |
| dog M2413 | AAV8.TBG.EGFP (bGH/+WPRE) | 3×1012 GC/kg | cephalic vein | 1.5 months | 7 days |
| dog M2396 | AAV8.TBG.EGFP (bGH/+WPRE) | 3×1012 GC/kg | cephalic vein | 1.5 months | 7 days |
| dog S473 | AAV8.TBG.EGFP (bGH/+WPRE) | 1.82×1013 GC/kg | hepatic artery | adult | 7 days |
| cynomolgus macaque C13991 | AAV8.TBG.EGFP (bGH/+WPRE) | 3×1012 GC/kg | saphenous vein | adult | 7 days |
| cynomolgus macaque C13992 | AAV8.TBG.EGFP (bGH/+WPRE) | 3×1012 GC/kg | saphenous vein | adult | 7 days |
| cynomolgus macaque 24466 | scAAV7.CB.EGFP (bGH/−WPRE) | 3.5×1012 GC/kg | saphenous vein | adult | 8 days |
| cynomolgus macaque 24456 | scAAV7.CB.EGFP (SV40/−WPRE) | 2×1012 GC/kg | saphenous vein | adult | 8 days |
| rhesus macaque 03D099 | AAV8.TBG.EGFP (bGH/+WPRE) | 3×1012 GC/kg | saphenous vein | adult | 7 days |
| rhesus macaque 607213 | AAV8.TBG.EGFP (bGH/+WPRE) | 3×1012 GC/kg | saphenous vein | adult | 7 days |
| rhesus macaque RQ8082 | AAV8.TBG.EGFP (bGH/+WPRE) | 3×1012 GC/kg | saphenous vein | juvenile (2-3 years) | 7 days |
| rhesus macaque RQ8083 | AAV8.TBG.EGFP (bGH/+WPRE) | 3×1012 GC/kg | saphenous vein | juvenile (2-3 years) | 7 days |
| infant rhesus macaque N1 | AAV8.TBG.EGFP (bGH/+WPRE) | 3×1012 GC/kg | peripheral vessel | 1 week | 7 days |
| infant rhesus macaque N5 | AAV8.TBG.EGFP (bGH/+WPRE) | 3×1012 GC/kg | peripheral vessel | 1 week | 35 days |
| infant rhesus macaque N7 | AAV8.TBG.EGFP (bGH/+WPRE) | 3×1012 GC/kg | peripheral vessel | 1 month | 45 days |