Human β -Globin Gene Transfer and Expression in Peripheral Blood
| Exp. No. . | Total Mice . | Short Term (<4 mo) . | Long Term (>4 mo) . | ||||
|---|---|---|---|---|---|---|---|
| (titer, cell source, cation) . | . | Mice PCR+ . | Mice RNA+ . | Hu β/Mouse β . | Mice PCR+ . | Mice RNA+ . | Hu β/Mouse β . |
| 1 (105, BM, PB) | 5 | 3 | NA | — | 3 | 2 | 20%* |
| 5%* | |||||||
| 2 (104, BM, PB) | 9 | 1 | 0 | — | 1 | 0 | — |
| 3 (105, FL, PB) | 10 | 4 | 1 | 3% | 1 | 0 | — |
| 4 (105, BM, PT) | 9 | 9 | 0 | — | 7 | 0 | — |
| Exp. No. . | Total Mice . | Short Term (<4 mo) . | Long Term (>4 mo) . | ||||
|---|---|---|---|---|---|---|---|
| (titer, cell source, cation) . | . | Mice PCR+ . | Mice RNA+ . | Hu β/Mouse β . | Mice PCR+ . | Mice RNA+ . | Hu β/Mouse β . |
| 1 (105, BM, PB) | 5 | 3 | NA | — | 3 | 2 | 20%* |
| 5%* | |||||||
| 2 (104, BM, PB) | 9 | 1 | 0 | — | 1 | 0 | — |
| 3 (105, FL, PB) | 10 | 4 | 1 | 3% | 1 | 0 | — |
| 4 (105, BM, PT) | 9 | 9 | 0 | — | 7 | 0 | — |
Hematopoietic cells were obtained, preincubated with growth factors, and then cocultured with retroviral producer cells. Lethally irradiated recipient mice were injected with transduced cells and peripheral blood samples were analyzed for gene transfer by PCR and gene expression by primer extension at various time points as described in Materials and Methods.
Abbreviations: Exp, experiment; PB, polybrene; PT, protamine; NA, not available.
Levels stable at 6 months and 8 months posttransplantation. These are mice 3 and 4 in Fig 4.