Table 1. Human cell engraftment in... | American Society of Hematology

Table 1.

Human cell engraftment in NOD/SCID/β2m^–/– and NOD/SCID mice and gene transfer efficiency ex vivo, in vivo, and in human CD34⁺ cells selected from mice

	% transgene⁺ cells ex vivo day 9	Human cell engraftment in BM, %	Transgene-positive engrafted human cells, %											% transgene⁺ in chimeric BM hCD34⁺ cultures
Group Mouse no.	% transgene⁺ cells ex vivo day 9	Human cell engraftment in BM, %			BM gp91^phox	BM CFP	Spleen CFP	PB gp91^phox	PB CFP	PB CFP	gp91phox	CFP	NBT (%)	CFP (%)
MF GS-CFP normal CD34⁺ PBSCs
I-4	97
I-4a		53	—	25	ND	—	ND	ND	—	10	ND	14/64 (22)
I-4b		46	—	21	ND	—	ND	ND	—	11	ND	4/46 (9)
II-4	87
II-4a		37	—	30	11	—	56	33	—	11	33/44 (75)	6/37 (16)
II-4b		27	—	30	9	—	77	50	—	6	22/29 (76)	1/38 (3)
II-4c		38	—	31	12	—	35	19	—	11	66/68 (97)	5/41 (12)
II-4d		40	—	31	13	—	52	34	—	11	57/60 (95)	3/29 (10)
III-A/B	97
III-A*		20	—	42	ND	—	60	ND	—	24	ND	12/54 (22)
III-B*		45	—	35	ND	—	53	ND	—	22	ND	4/33 (12)
III-C/D	98
III-C*		3	—	48	ND	—	ND	ND	—	ND	ND	ND
III-D*		7	—	42	ND	—	ND	ND	—	24	ND	ND
III-E/F	99
III-E*		8	—	67	ND	—	ND	ND	—	57	ND	ND
III-F*		0.5	—	77	ND	—	ND	ND	—	ND	ND	ND
IV	82
IV-A*		18	—	25	ND	—	ND	ND	—	ND	ND	ND
IV-B*		26	—	27	ND	—	ND	ND	—	ND	ND	ND
IV-C*		15	—	32	ND	—	ND	ND	—	ND	ND	ND
IV-D*		35	—	20	ND	—	ND	ND	—	ND	ND	ND
Naive X-CGD CD34⁺ PBSCs
I-2, II-2	< 0.5		< 1	< 1	< 2	< 2	< 2	< 0.5	< 0.3	< 0.3
I-2a		14									ND	—
I-2b		13									ND	—
II-2a		67									0/25	—
II-2b		55									0/12	—
II-2c		62									0/16	—
MFGS-gp91^phox X-CGD CD34⁺ PBSCs
I-3†	92	16	14	—	—	ND	—	—	7	—	ND	—
II-3	96
II-3a		36	22	—	—	25	—	—	10	—	3/28 (11)	—
II-3b		36	19	—	—	18	—	—	7	—	4/36 (11)	—
II-3c		31	19	—	—	16	—	—	7	—	3/34 (9)	—
II-3d		34	17	—	—	10	—	—	10	—	5/32 (16)	—
Column 1	2	3	4	5	6	7	8	9	10	11	12	13

	% transgene⁺ cells ex vivo day 9	Human cell engraftment in BM, %	Transgene-positive engrafted human cells, %											% transgene⁺ in chimeric BM hCD34⁺ cultures
Group Mouse no.	% transgene⁺ cells ex vivo day 9	Human cell engraftment in BM, %			BM gp91^phox	BM CFP	Spleen CFP	PB gp91^phox	PB CFP	PB CFP	gp91phox	CFP	NBT (%)	CFP (%)
MF GS-CFP normal CD34⁺ PBSCs
I-4	97
I-4a		53	—	25	ND	—	ND	ND	—	10	ND	14/64 (22)
I-4b		46	—	21	ND	—	ND	ND	—	11	ND	4/46 (9)
II-4	87
II-4a		37	—	30	11	—	56	33	—	11	33/44 (75)	6/37 (16)
II-4b		27	—	30	9	—	77	50	—	6	22/29 (76)	1/38 (3)
II-4c		38	—	31	12	—	35	19	—	11	66/68 (97)	5/41 (12)
II-4d		40	—	31	13	—	52	34	—	11	57/60 (95)	3/29 (10)
III-A/B	97
III-A*		20	—	42	ND	—	60	ND	—	24	ND	12/54 (22)
III-B*		45	—	35	ND	—	53	ND	—	22	ND	4/33 (12)
III-C/D	98
III-C*		3	—	48	ND	—	ND	ND	—	ND	ND	ND
III-D*		7	—	42	ND	—	ND	ND	—	24	ND	ND
III-E/F	99
III-E*		8	—	67	ND	—	ND	ND	—	57	ND	ND
III-F*		0.5	—	77	ND	—	ND	ND	—	ND	ND	ND
IV	82
IV-A*		18	—	25	ND	—	ND	ND	—	ND	ND	ND
IV-B*		26	—	27	ND	—	ND	ND	—	ND	ND	ND
IV-C*		15	—	32	ND	—	ND	ND	—	ND	ND	ND
IV-D*		35	—	20	ND	—	ND	ND	—	ND	ND	ND
Naive X-CGD CD34⁺ PBSCs
I-2, II-2	< 0.5		< 1	< 1	< 2	< 2	< 2	< 0.5	< 0.3	< 0.3
I-2a		14									ND	—
I-2b		13									ND	—
II-2a		67									0/25	—
II-2b		55									0/12	—
II-2c		62									0/16	—
MFGS-gp91^phox X-CGD CD34⁺ PBSCs
I-3†	92	16	14	—	—	ND	—	—	7	—	ND	—
II-3	96
II-3a		36	22	—	—	25	—	—	10	—	3/28 (11)	—
II-3b		36	19	—	—	18	—	—	7	—	4/36 (11)	—
II-3c		31	19	—	—	16	—	—	7	—	3/34 (9)	—
II-3d		34	17	—	—	10	—	—	10	—	5/32 (16)	—
Column 1	2	3	4	5	6	7	8	9	10	11	12	13

Data in columns 2 to 11 were derived from flow cytometric analysis. Analysis was performed without gating or was limited to gates as indicated in the columns: human granulocyte gate (CD45⁺ CD13⁺, high side scatter [SSC]) for columns 4, 5, 7, and 8 or human B-cell gate (CD45⁺/CD19⁺) for columns 6 and 9; data in columns 10 to 13 were derived from cultures of human CD34⁺ cells isolated from chimeric mouse BM (liquid culture for columns 10 and 11; semisolid colony-forming unit [CFU] assays for columns 12 and 13). Data in columns 12 and 13 were derived by microscopic analysis. Mice III-C, III-D and mice III-E, III-F received CD34⁺ PBSCs cultured and transduced for 5 and 6 days, respectively. All other mice received transplants of 4-day cultured cells. Mice were killed after 6 weeks, except group IV, which was killed after 9 weeks. Background values for human CD45 staining in mice not receiving transplants (not shown) were < 2% and were subtracted from the given human engraftment levels. Similarly, background levels from cells that served as negative control were subtracted from values indicated for transduced cells. —, indicates not applicable; and ND, not done.

The 10 mice were NOD/SCID mice; all others were NOD/SCID/β2m^-/- mice.

†

There is only one mouse in group I-3.

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