Table 2. Therapeutic efficacy of the insulated γ-globin vector in sickle cell disease mice given subablative conditioning.
| Transplant group | n | Hb (g/dl) | RBC (×106/µl) | WBC (×103/µl) | ANC (×109/µl) | Urine mosm/kg | Spleen (g) |
|---|---|---|---|---|---|---|---|
| Mock | 18 | 7.7 ± 1.2 | 6.1 ± 0.7 | 23.7 ± 8.0 | 4.8 ± 2.4 | 1,529 ± 433 | 1.5 ± 0.5 |
| V5m3-400 | 21 | 8.9 ± 0.9 | 7.5 ± 0.7 | 13.1 ± 4.2 | 3.0 ± 1.3 | 2,687 ± 479 | 0.9 ± 0.3 |
| WT Bl6 | 14 | 13 ± 0.4 | 9.3 ± 0.4 | 7.4 ± 1.3 | 1.3 ± 0.3 | 3,370 ± 256a | 0.1 ± 0.04a |
Values represent the mean and standard deviation (SD). All data were obtained 4–5 months after transplantation.
ANC, absolute neutrophil count; Hb, total hemoglobin concentration; RBC, red blood cell; WBC, white blood cell.
a
WT Bl6 urine concentrating ability and spleen size determined for (n = 5 mice).