Table 1 Phenotypic correction of... | American Society of Hematology

Table 1

Phenotypic correction of BAL-fVIIIKO hemophilia A mice

Mouse	Conditioning	Vector	Weeks after transplantation	% GFP⁺	fVIII, ng/mL	fVIII, mIU/mL	Survival
Primaries
1	550 cGy	MSGV-sfVIIIΔB	24	14.3	27.2	181	+
2	550 cGy	MSGV-sfVIIIΔB	24	19.3	35.9	153	+
3	550 cGy	MSGV-sfVIIIΔB	24	35.4	57.6	339	+
4	550 cGy	MSGV-sfVIIIΔB	24	29.8	44.3	256	+
5	550 cGy	FB.efVIII	24	NA	59.3	292	+
6	550 cGy	FB.efVIII	24	NA	31	201	+
7	550 cGy	FB.efVIII	24	NA	25.5	177	+
8	550 cGy	FB.efVIII	24	NA	17.3	218	+
9	550 cGy	FB.efVIII	24	NA	49.8	407	+
10	550 cGy	FB.efVIII	24	NA	47.8	231	+
11	BU-ATS	MSGV-sfVIIIΔB	24	11.3	41.1	86	+
12	BU-ATS	MSGV-sfVIIIΔB	24	14.4	38.9	337	+
13	BU-ATS	MSGV-sfVIIIΔB	24	21.4	42.7	241	+
14	BU-ATS	MSGV-sfVIIIΔB	24	33.9	25.6	181	+
15	BU-ATS	MSGV-sfVIIIΔB	24	15.7	27.7	123	+
16	BU-ATS	MSGV-sfVIIIΔB	24	9.6	31.4	189	+
17	BU-ATS	MSGV-sfVIIIΔB	12	12.5	49.5	335	ND
18	BU-ATS	MSGV-sfVIIIΔB	8	21.5	36.6	184	ND
19	BU-ATS	FB.efVIII	24	NA	52.2	143	+
20	BU-ATS	FB.efVIII	24	NA	33.4	111	+
21	BU-ATS	FB.efVIII	24	NA	31.9	172	+
22	BU-ATS	FB.efVIII	24	NA	28.9	151	+
23	BU-ATS	FB.efVIII	24	NA	44.1	277	+
24	BU-ATS	FB.efVIII	24	NA	53.7	133	+
25	BU-ATS	FB.efVIII	24	NA	39.8	255	+
26	BU-ATS	FB.efVIII	24	NA	29.1	103	+
Secondaries
1	800 cGy	MSGV-sfVIIIΔB	16	9.1	25.3	121	+
2	800 cGy	MSGV-sfVIIIΔB	16	5.4	18.6	73	+
3	800 cGy	FB.efVIII	16	NA	14.8	128	+
4	800 cGy	FB.efVIII	16	NA	19.4	177	+
5	800 cGy	MSGV-sfVIIIΔB	16	8.2	21.7	155	+
6	800 cGy	MSGV-sfVIIIΔB	16	7	11.9	81	+
7	800 cGy	FB.efVIII	16	NA	13.5	59	+
8	800 cGy	FB.efVIII	16	NA	16.3	126	+
Controls
BAL-fVIIIKO							0/5
Wild type							5/5

Mouse	Conditioning	Vector	Weeks after transplantation	% GFP⁺	fVIII, ng/mL	fVIII, mIU/mL	Survival
Primaries
1	550 cGy	MSGV-sfVIIIΔB	24	14.3	27.2	181	+
2	550 cGy	MSGV-sfVIIIΔB	24	19.3	35.9	153	+
3	550 cGy	MSGV-sfVIIIΔB	24	35.4	57.6	339	+
4	550 cGy	MSGV-sfVIIIΔB	24	29.8	44.3	256	+
5	550 cGy	FB.efVIII	24	NA	59.3	292	+
6	550 cGy	FB.efVIII	24	NA	31	201	+
7	550 cGy	FB.efVIII	24	NA	25.5	177	+
8	550 cGy	FB.efVIII	24	NA	17.3	218	+
9	550 cGy	FB.efVIII	24	NA	49.8	407	+
10	550 cGy	FB.efVIII	24	NA	47.8	231	+
11	BU-ATS	MSGV-sfVIIIΔB	24	11.3	41.1	86	+
12	BU-ATS	MSGV-sfVIIIΔB	24	14.4	38.9	337	+
13	BU-ATS	MSGV-sfVIIIΔB	24	21.4	42.7	241	+
14	BU-ATS	MSGV-sfVIIIΔB	24	33.9	25.6	181	+
15	BU-ATS	MSGV-sfVIIIΔB	24	15.7	27.7	123	+
16	BU-ATS	MSGV-sfVIIIΔB	24	9.6	31.4	189	+
17	BU-ATS	MSGV-sfVIIIΔB	12	12.5	49.5	335	ND
18	BU-ATS	MSGV-sfVIIIΔB	8	21.5	36.6	184	ND
19	BU-ATS	FB.efVIII	24	NA	52.2	143	+
20	BU-ATS	FB.efVIII	24	NA	33.4	111	+
21	BU-ATS	FB.efVIII	24	NA	31.9	172	+
22	BU-ATS	FB.efVIII	24	NA	28.9	151	+
23	BU-ATS	FB.efVIII	24	NA	44.1	277	+
24	BU-ATS	FB.efVIII	24	NA	53.7	133	+
25	BU-ATS	FB.efVIII	24	NA	39.8	255	+
26	BU-ATS	FB.efVIII	24	NA	29.1	103	+
Secondaries
1	800 cGy	MSGV-sfVIIIΔB	16	9.1	25.3	121	+
2	800 cGy	MSGV-sfVIIIΔB	16	5.4	18.6	73	+
3	800 cGy	FB.efVIII	16	NA	14.8	128	+
4	800 cGy	FB.efVIII	16	NA	19.4	177	+
5	800 cGy	MSGV-sfVIIIΔB	16	8.2	21.7	155	+
6	800 cGy	MSGV-sfVIIIΔB	16	7	11.9	81	+
7	800 cGy	FB.efVIII	16	NA	13.5	59	+
8	800 cGy	FB.efVIII	16	NA	16.3	126	+
Controls
BAL-fVIIIKO							0/5
Wild type							5/5

NA indicates not applicable; and ND, not done.