Inhibition of AAV2-mediated gene transfer to mouse liver by neutralizing antibodies
Neutralizing antibody titer range . | FIX DNA copy/diploid genome,*mean ± SD . | FIX levels, ng/mL,†mean ± SD . |
|---|---|---|
| 0 | 1.04 ± 0.698 | 13 286 ± 6 521 |
| 1:4–1:12.5 | 0.008 ± 0.017 | 17 ± 46 |
| 1:33–1:100 | < 0.0003 | < 15 |
Neutralizing antibody titer range . | FIX DNA copy/diploid genome,*mean ± SD . | FIX levels, ng/mL,†mean ± SD . |
|---|---|---|
| 0 | 1.04 ± 0.698 | 13 286 ± 6 521 |
| 1:4–1:12.5 | 0.008 ± 0.017 | 17 ± 46 |
| 1:33–1:100 | < 0.0003 | < 15 |
Determined by Q-PCR of transgene FIX DNA in mouse liver tissues harvested at 12 weeks after vector injection. N = 7 mice per group. The limit of detection is 0.0003 copy per diploid genome
Determined by ELISA for human FIX in mouse plasmas collected at 4 weeks after vector injection. N = 7 mice per group. The limit of detection is 15 ng/mL. The normal human FIX level is 5000 ng/mL