Determination of vector genome copy number after ATVRX delivery of an AAV2-cFIX vector
| Biopsy site . | DLAM 3, vg/diploid genome . | DLAM 4, vg/diploid genome . |
|---|---|---|
| Target limb | ||
| 1 | 0.163 ± 0.111 | 0.09* |
| 2 | 0.091 ± 0.012 | 0.186 ± 0.251 |
| 3 | 14.018 ± 1.479 | 9.146 ± 1.587 |
| 4 | 6.058 ± 0.591 | 0.505 ± 0.057 |
| 5 | 0.019 ± 0.005 | 5.358 ± 0.592 |
| Contralateral limb | ||
| 1 | 0.004 ± 0.001 | |
| 2 | 0.014 ± 0.015 | 0.094 ± 0.108 |
| 3 | 0.004 ± 0.003 | |
| 4 | 0.004 ± 0.001 | |
| 5 | 0.004 ± 0.004 | 0.012 ± 0.003 |
| Biopsy site . | DLAM 3, vg/diploid genome . | DLAM 4, vg/diploid genome . |
|---|---|---|
| Target limb | ||
| 1 | 0.163 ± 0.111 | 0.09* |
| 2 | 0.091 ± 0.012 | 0.186 ± 0.251 |
| 3 | 14.018 ± 1.479 | 9.146 ± 1.587 |
| 4 | 6.058 ± 0.591 | 0.505 ± 0.057 |
| 5 | 0.019 ± 0.005 | 5.358 ± 0.592 |
| Contralateral limb | ||
| 1 | 0.004 ± 0.001 | |
| 2 | 0.014 ± 0.015 | 0.094 ± 0.108 |
| 3 | 0.004 ± 0.003 | |
| 4 | 0.004 ± 0.001 | |
| 5 | 0.004 ± 0.004 | 0.012 ± 0.003 |
Results are expressed as average ± SD. All the experiments were repeated twice using 500 to 1000 ng of genomic DNA per reaction.
Single experiment with 344 ng of genomic DNA in the reaction.