Table 2.

FICD-transduced cells perform in NOD∕SCID mice

Patient no.	Human BM FICD-transduced cells				Mice used	NOD∕SCID mice assay
	Day 0		Day 63			BM				Spleen colonies	Liver colonies
	Cell count	CD34+ %	Cell count	CD34+ %		Colonies	CFU-readout
							Erythroid	Myeloid	GEMM
5. Cha	2 × 105	5	2 × 104	20	1	185	3 BFU-E, 4 CFU-E	176	2	71 (21 clusters)	0
7. Cas	2 × 105	1	3.05 × 105	18	2	0	—	—	—	0	0
9. OCT	2 × 105	0.50	3.2 × 105	25	2	13	—	6 GM, 4 M, 3 G	—	0	0

hCFU assay from NOD∕SCID mice BM samples, 14 weeks (over 3 months) after infusion of transduced cells primarily grown in LTC for 9 weeks; CD34⁺ percentages were measured before infusion. Human CFUs were obtained only with cells from the early diagnosed patient.