TABLE 1.
Summary of results from high-throughput rAAV integration site analysis
| Mouse | Vector | Vector dose (1010) | Routeb | Tissue | No. of clones in each category
|
Efficiency (%)a | |||
|---|---|---|---|---|---|---|---|---|---|
| Total sequenced | Mouse hit | rRNA gene repeat | Mapped to a unique site | ||||||
| B6 | AAV8 | 720 | TV | Liver | 151 | 25 | 1 | 24 | 16.6 |
| B6 | AAV8 | 5 | TV | Liver | 230 | 218 | 11 | 201 | 94.8 |
| B6 | AAV2 | 30 | PV | Liver | 84 | 80 | 2 | 76 | 95.2 |
| B6 | AAV8 | 720 | TV | Muscle | 21 | 17 | 1 | 16 | 81.0 |
| B6 | AAV8 | 720 | TV | Heart | 93 | 86 | 1 | 83 | 92.5 |
| B6 total | 579 | 426 | 16 | 400 | 73.6 | ||||
| SCID | AAV8 | 720 | TV | Liver | 229 | 166 | 5 | 160 | 72.5 |
| SCID | AAV8 | 5 | TV | Liver | 74 | 65 | 0 | 63 | 87.8 |
| SCID | AAV2 | 30 | PV | Liver | 86 | 82 | 2 | 79 | 95.3 |
| SCID | AAV8 | 720 | TV | Muscle | 189 | 161 | 5 | 148 | 85.2 |
| SCID | AAV8 | 720 | TV | Heart | 112 | 97 | 1 | 91 | 86.6 |
| SCID total | 690 | 571 | 13 | 541 | 82.8 | ||||
| Total | 1,269 | 997 | 29 | 941 | 78.6 | ||||
a
Efficiency is derived from the following calculation: (number of clones containing a sequence from the mouse genome)/(number of total clones sequenced) × 100.
b
TV, tail vein; PV, portal vein.