Table 3. Human FVIII expression at 6... | American Society of Hematology

Table 3.

Human FVIII expression at 6 weeks after injection of Hem-A/Rag1 mice

Vector	Mouse	FVIII-LC antigen, ng/mL	FVIII-HC antigen, ng/mL	FVIII activity, mU/mL	Specific activity, U/mg FVIII LC	Specific activity, U/mg FVIII HC
HC and LC	1.1	1566	98	330	210	3367
	1.2	2102	42	222	106	5285
	2.0	414	< 15.6	< 50	0	0
	2.1	1298	65	453	349	6969
	2.2	3242	Not done	559	172	Not done
	3.0	977	33	76	77	2303
LC	3.1	1848	< 15.6	< 50	0	0
	3.2	4180	< 15.6	< 50	0	0
HC	4.2	< 156	18	< 50	0	0
	4.1	< 156	24	< 50	0	0

Vector	Mouse	FVIII-LC antigen, ng/mL	FVIII-HC antigen, ng/mL	FVIII activity, mU/mL	Specific activity, U/mg FVIII LC	Specific activity, U/mg FVIII HC
HC and LC	1.1	1566	98	330	210	3367
	1.2	2102	42	222	106	5285
	2.0	414	< 15.6	< 50	0	0
	2.1	1298	65	453	349	6969
	2.2	3242	Not done	559	172	Not done
	3.0	977	33	76	77	2303
LC	3.1	1848	< 15.6	< 50	0	0
	3.2	4180	< 15.6	< 50	0	0
HC	4.2	< 156	18	< 50	0	0
	4.1	< 156	24	< 50	0	0

Efficacy of AAV-hFVIII vectors in vivo at 6 weeks after injection. Hem-A/Rag1 mice were injected with 3 × 10¹¹ vg/mouse of both AAV-hFVIII-HC and AAV-hFVIII-LC or AAV-hFVIII-LC alone or AAV-hFVIII-HC alone. FVIII-LC and HC antigen levels were determined by antigen-specific ELISAs. FVIII activity was determined by Coatest. Specific activity levels based on both the heavy and light chains were separately determined.

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