Improved expression and efficacy of the mLARβΔγV5 vector in murine β-thalassemia
Transplant group . | n . | % F* . | % F cells† . | Vector copy‡ . | Hb, g/L . | % Reticulocytes . | Spleen weight, mg . |
|---|---|---|---|---|---|---|---|
| Mock | 5 | NA | NA | NA | 86 ± 3 | 22 ± 3.4 | 205 ± 21 |
| d432βΔγm | 5 | 8.4 ± 2 | 31 ± 5 | 2.0 ± 0.3 | 103 ± 3§ | 17.3 ± 1.0∥ | 159 ± 7¶ |
| mLARβΔγV5 | 6 | 22 ± 4♯ | 71 ± 10♯ | 1.3 ± 0.3** | 112 ± 3§†† | 6.4 ± 0.7§♯ | 115 ± 8§♯ |
| Normal control | 5 | NA | NA | NA | 122 ± 02§ | 4.0 ± 0.3§ | ND |
Transplant group . | n . | % F* . | % F cells† . | Vector copy‡ . | Hb, g/L . | % Reticulocytes . | Spleen weight, mg . |
|---|---|---|---|---|---|---|---|
| Mock | 5 | NA | NA | NA | 86 ± 3 | 22 ± 3.4 | 205 ± 21 |
| d432βΔγm | 5 | 8.4 ± 2 | 31 ± 5 | 2.0 ± 0.3 | 103 ± 3§ | 17.3 ± 1.0∥ | 159 ± 7¶ |
| mLARβΔγV5 | 6 | 22 ± 4♯ | 71 ± 10♯ | 1.3 ± 0.3** | 112 ± 3§†† | 6.4 ± 0.7§♯ | 115 ± 8§♯ |
| Normal control | 5 | NA | NA | NA | 122 ± 02§ | 4.0 ± 0.3§ | ND |
Values represent the mean and SEM.
Hb indicates hemoglobin concentration; NA, not applicable; and ND, not done.
Amount of mα2, hγ2 present in the peripheral blood as a percentage of the total Hb.
Percentage of red cells staining for γ-globin expression as determined by FACS analysis.
Average vector copy number in PB leukocytes as determined by semiquantitative DNA PCR.
Statistically significant difference from mock group with P at < .01, one-sided.
Not statistically different from mock with P = .11, one-sided.
Statistically different from mock group with P < .05, one-sided.
Statistically significant difference from d432βΔγm group with P at least < .02, one-sided.
Not significantly different from d432βΔγm group with P = .098, 2-sided.
Statistically significant difference from d432βΔγm group with P < .03, one-sided.