Table 1.

Hypoxia and NAC conditioning protects FA-A patients’ BM against initial cell damage

Patient no.	Cell counts						CFU assays§							Vector∥
	Day 0*		Day 2†		Day 8‡		Erythroid		Myeloid		GEMM	Total¶
	Per ml BM	Percent CD34+	Conditioning				Conditioning							FICD	Lenti
			No	Yes	No	Yes	No	Yes	No	Yes	Yes	No	Yes
1. Ale	2.36 × 106	1.34	ND	ND	27,000	208,000	0	8	0	55	0	0	63	+	−
2. Jav	7.4 × 106	0.3	Debris	140,000	Debris	72,000	0	121	0	48	3	0	172	+	+
3. Jul	1.9 × 106	ND	ND	150,000	ND	56,000	0	2	0	15	0	0	17	+	+
4. Aud	1.3 × 106	ND	ND	150,000	ND	93,000	0	1	0	17	1	0	19	+	−
5. Cha	4.9 × 106	5	288,000	308,000	14,000	147,000	0	14	0	37	0	0	51	+	+
6. Lau	0.7 × 106	0.9	54,000	66,000	ND	ND	0	2	5	13	0	5	15	+	+
7. Cas**	2 × 106	1	1,206,000	1,562,000	80,000	116,000	0	1	5	13	1	5	15	+	−
8. Rho**	1.9 × 106	10.2	468,000	374,000	4,000	36,000	0	0	1 cluster	12 cluster	0	1	12	+	+
9. OCT**	0.4 × 106	0.5	1,188,000	1,518,000	6,800	124,000	6	5	11	26	1	17	32	+	−
p value					0.045		0.027					0.009

*BM sample cell concentration and percentage of CD34⁺.

^†BM cells counts starting from 2 × 10⁵ cells and expressed per ml, with or without conditioning.

^‡The effects of conditioning on cell survival appear to be statistically significant (p = 0.045) at day 8.

^§CFU assays (from 50 × 10³ at most) performed with or without NAC and hypoxia conditioning. Erythroid colonies grew from eight conditioned patients’ samples and only from one unconditioned (9) (p = 0.027). Myeloid colonies readout is monitored in all conditioned samples and mix (CFU-GEMM) colonies grow from these only (2, 4, 7, and 9) because neither BFU-E nor GEMM-CFU can be observed out of unconditioned samples.

^¶The overall p value (0.009) is highly significant (Wilcoxon paired test).

^∥Indication of which patients’ samples have been exposed to both FICD and lentivirus-based vectors in the transduction experiments.

**Cultures from patients 7, 8, and 9 were initiated from 1 × 10⁶ cells.