Table 1.

Evaluation of autologous PBSC after ex vivo transduction and culture

Patient*	Prep	Cells infused†	% CD34+‡	% of normal chemiluminescence§	DHR assay,§ % granulocytes corrected	% NBT-positive colonies¶	Vector copy number‖
1	A	60	85	25	30	9	0.05
	B	200  (4.7)	97	26	21	6	0.11
2	A	12	84	26	90	29	0.19
	B	45  (0.9)	94	23	44	28	0.08
3	A	215	61	65	75	14	0.13
	B	66  (4.3)	85	64	75	18	0.18
4	A	77	92	27	34	9	0.16
	B	129  (2.5)	81	36	63	11	0.11
5	A	2	63	32	27	19	0.13
	B	2  (0.1)	79	39	59	14	0.13

^*Each patient received by vein two preparations (Prep, A and B) of the transduced and cultured PBSCs derived from the first and second apheresis procedures, respectively. 

^†At culture day 3 for each preparation, the number of cells shown (×10⁻⁶) were infused intravenously. Shown in the parentheses is the total number of cells (×10⁻⁶) (A plus B) infused per kg of body weight. 

^‡Measured by flow cytometry analysis at the end of culture day 3. 

^§Assays were performed on day 17 of culture. 

^¶Cells were plated at culture day 3 and assayed 14 days later. 

^‖Measured by Southern blot of genomic DNA from the transduced and cultured PBSCs, probed with a MFGS-vector-specific 5′ long terminal repeat sequence and a cell line with known vector copy number of 1 as a reference.