Table 1.
Human β-globin expression, Hb levels, and VCN in th3/+ mice transplanted with transduced BM
| Mouse no. | Peripheral blood |
BM |
CFU-S |
|||
|---|---|---|---|---|---|---|
| % huβ+ RBCs | % huβ/mα* | Hb, g/dl | VCN | % vector+ | VCN | |
| 14 | 64 | ND | 9.69 | 0.23 | 35 | 1.06 ± 0.10 |
| 19 | 99 | 19 | 12.48 | 0.91 | 57 | 1.11 ± 0.12 |
| 21 | 95 | 19 | 14.01 | 0.74 | ND | ND |
| 4 | 99 | 23 | 12.30 | 0.55 | 45 | 1.18 ± 0.08 |
| 6 | 86 | 14 | 9.40 | 0.27 | ND | ND |
| 5 | 99 | 37 | 13.86 | 0.83 | ND | ND |
| 2 | 95 | 30 | 13.20 | 2.20 | 100 | 3.77 ± 0.51 |
| 55 | 96 | ND | 11.65 | 0.55 | 63 | 1.17 ± 0.10 |
CFU-S, spleen colony-forming units; ND, not determined.
*Ratio between the areas of HPLC peaks corresponding to human β - and murine α-globin chains (huβ/mα).