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. 2013 Jan 31;121(21):4396–4403. doi: 10.1182/blood-2012-10-464164

Table 1.

Summary of gene transfer and expression results for mice receiving AAV8-FVIII-BDD or AAV8-FVIII-RH

Dose Gene copy number (copies per cell) RNA transcripts (FVIII/18S RNA) Circulating FVIII (ng/mL)
AAV8-BDD AAV8-RH AAV8-BDD AAV8-RH AAV8-BDD AAV8-RH
8 × 1012 vg/kg 16.8 ± 4.2 18.7 ± 4.0 0.09 ± 0.03 0.11 ± 0.04 49.9 ± 9.4 78.9 ± 6.6
2 × 1013 vg/kg 64.6 ± 15.6 35.0 ± 9.1 0.04 ± 0.01 0.09 ± 0.03 71.4 ± 7.5 123.5 ± 19.3
4 × 1013 vg/kg 84.7 ± 24.0 71.4 ± 12.9 0.10 ± 0.06 0.23 ± 0.12 75.1 ± 9.8 158.1 ± 28.3
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