Table 1 Summary of gene transfer and... | American Society of Hematology

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)
Dose	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

Dose	Gene copy number (copies per cell)		RNA transcripts (FVIII/18S RNA)		Circulating FVIII (ng/mL)
Dose	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