Table 1.
Summary of the control and multicopy lines generated, transgene copy numbers, γ-globin mRNA expression levels in adult blood, and integration sites of the transgene.
| Transgenic lines | Copy no. | % γ/βmaj Globina | % γ/βmaj Globin per copya | Chromosomal integration site |
|---|---|---|---|---|
| HPFH2C 16.5 dpc embryo | 1 | 26.33 ± 4.73 | 52.67 ± 9.45 | Euchromatic |
| HPFH2C adult | 1 | 2.46 ± 0.93 | 4.91 ± 1.87 | Euchromatic |
| LCRAγ-C | 1 | 0 | 0 | Euchromatic |
| LCRAγ-F | 1 | 0 | 0 | Centromeric |
| LCRAγ-H | 1 | 0 | 0 | Euchromatic |
| LCR-Aγ-ΔEnh/F-1 | 2 | 16.40 ± 3.86 | 16.40 ± 3.86 | Centromeric |
| LCR-Aγ-ΔEnh/F-2 | 3 | 19.97 ± 3.95 | 13.31 ± 2.63 | Euchromatic |
| LCR-Aγ-ΔEnh/F-3 | 4 | 35.57 ± 1.25 | 17.78 ± 0.62 | Euchromatic |
a
Expression of the fetal Aγ-gene was calculated by S1 nuclease analysis as 1) a percentage of total βmaj globin expression and 2) per transgene copy and per copy of the endogenous βmaj mouse globin gene. Mean and standard deviations are derived from three independent RNA samples.