Long-term engraftment in vivo in NOD SCID mice
| Experiment . | No. mice (mo post-BMT) . | No. CD34+/mouse . | % huCD45 in BM . | Avg VCN bulk BM . | Frequency vector +ve BFU-E/BM (%) . | β/α** Expression BFU-E/BM (%) . |
|---|---|---|---|---|---|---|
| Postinfusion (in vivo) | ||||||
| 1 | 1 (6) | 5.0 × 105 | 54 | 0.17 | 36.0 | ND |
| 2 | 6 (7) | 1.8 × 106 | 13.6 ± 9.8 | 0.50 ± 0.3 | 31.0 | 77.8 ± 55 |
| 3 | 3 (6) | 2.0 × 106 | 0.8 ± 0.2 | 0.54 ± 0.2 | ND | ND |
| 4 | 3 (6) | 6.7 × 105 | 2.5 ± 2.6 | 0.60 ± 0.9 | ND | ND |
| 5 | 4 (3) | 1.6 × 106 | 3.2 ± 4.6 | 0.60 ± 0.3 | ND | ND |
| Experiment . | No. mice (mo post-BMT) . | No. CD34+/mouse . | % huCD45 in BM . | Avg VCN bulk BM . | Frequency vector +ve BFU-E/BM (%) . | β/α** Expression BFU-E/BM (%) . |
|---|---|---|---|---|---|---|
| Postinfusion (in vivo) | ||||||
| 1 | 1 (6) | 5.0 × 105 | 54 | 0.17 | 36.0 | ND |
| 2 | 6 (7) | 1.8 × 106 | 13.6 ± 9.8 | 0.50 ± 0.3 | 31.0 | 77.8 ± 55 |
| 3 | 3 (6) | 2.0 × 106 | 0.8 ± 0.2 | 0.54 ± 0.2 | ND | ND |
| 4 | 3 (6) | 6.7 × 105 | 2.5 ± 2.6 | 0.60 ± 0.9 | ND | ND |
| 5 | 4 (3) | 1.6 × 106 | 3.2 ± 4.6 | 0.60 ± 0.3 | ND | ND |
Mice were infused with 5.0e5 to 2.0e6 transduced CD34+ HPCs. The average VCN and the frequency of BFU-E colonies positive for the vector were determined as well as the level of β/α chain expression in the BFU-Es derived from the BM of engrafted mice (BFU-E/BM β/α** expression was measured in 2 independent pools of transduced BFU-Es and averaged. Untransduced pool 1, n = 30; transduced with TNS9.3.55 pool 1, n = 32; average VCN = 1.2. Untransduced pool 2, n = 30; transduced with TNS9.3.55 pool 2, n = 30; average VCN = 1.1. Experiments 4 and 5 were conducted with TNS9.3.55-transduced CD34+ cells derived from validation 2 and validation 3, respectively. CFU-GM, granulocyte-macrophage CFU; ND, not done; +ve, positive.