Table 1. Gene copy number and transgene... | American Society of Hematology

Table 1.

Gene copy number and transgene expression in C57BI/6 Rag-1 mice 14 weeks after intramuscular injection of AAV vector encoding F.IX-WT or 1 K5A/V10K

	Vector genomes/cell			F.IX levels, ng/mL
Vector dose, vg/kg	K5A/V10K	WT	K5A/V10K	WT	K5A/V10K	WT
2 × 10¹¹	5.9 ± 2.8	9.3 ± 4.7	451 ± 130^*	209 ± 109	76.4 ± 18^*	22.4 ± 5
1.2 × 10¹²	21.6 ± 8.2	21.9 ± 6.3	1670 ± 360†	403 ± 96	77.3 ± 24^*	18.4 ± 12

	Vector genomes/cell			F.IX levels, ng/mL
Vector dose, vg/kg	K5A/V10K	WT	K5A/V10K	WT	K5A/V10K	WT
2 × 10¹¹	5.9 ± 2.8	9.3 ± 4.7	451 ± 130^*	209 ± 109	76.4 ± 18^*	22.4 ± 5
1.2 × 10¹²	21.6 ± 8.2	21.9 ± 6.3	1670 ± 360†	403 ± 96	77.3 ± 24^*	18.4 ± 12

Student t test was used for comparison between F.IX-WT and K5A/V10K: ^*P < .05; †P < .005.

N = 4 mice per vector per dose. Data presented are mean values ± SEM.

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