Improvement of β-thalassemic red blood cell indices by lentiviral-mediated human γ-globin expression
| Transplant group . | n . | % F* . | % F cells† . | Vector copy‡ . | Hb level, g/dL . | MCV, fL . | MCH level, pg . | RDW . | Retic count, % . |
|---|---|---|---|---|---|---|---|---|---|
| Mock | 8 | NA | NA | NA | 9.1 ± 0.1 | 41.6 ± 0.4 | 10.3 ± 0.2 | 30.2 ± 0.5 | 25.2 ± 0.6 |
| Less than 4% F | 7 | <4% | 21.1 ± 3.6 | 0.8 ± 0.2 | 9.4 ± 0.1 | 41.9 ± 0.3 | 11.0 ± 0.1# | 29.3 ± 0.5 | 27.5 ± 1.2 |
| 10% to 15% F | 9 | 12.6 ± 0.5 | 61.9 ± 5.3 | 2.1 ± 0.2 | 10.1 ± 0.1# | 44.9 ± 0.3# | 11.3 ± 0.2# | 29.1 ± 0.4 | 20.8 ± 0.9# |
| 15% or more F | 5 | 20.8 ± 4.01-153 | 80.4 ± 6.61-155 | 2.4 ± 0.71-154 | 11.6 ± 0.31-153# | 48.0 ± 2.0# | 13.1 ± 0.7# | 25.7 ± 1.6# | 12.6 ± 2.8# |
| Normal control | 11 | NA | NA | NA | 12.8 ± 0.1# | 52.9 ± 0.9# | 14.8 ± 0.2# | 18.6 ± 0.3# | 3.3 ± 0.1# |
| Transplant group . | n . | % F* . | % F cells† . | Vector copy‡ . | Hb level, g/dL . | MCV, fL . | MCH level, pg . | RDW . | Retic count, % . |
|---|---|---|---|---|---|---|---|---|---|
| Mock | 8 | NA | NA | NA | 9.1 ± 0.1 | 41.6 ± 0.4 | 10.3 ± 0.2 | 30.2 ± 0.5 | 25.2 ± 0.6 |
| Less than 4% F | 7 | <4% | 21.1 ± 3.6 | 0.8 ± 0.2 | 9.4 ± 0.1 | 41.9 ± 0.3 | 11.0 ± 0.1# | 29.3 ± 0.5 | 27.5 ± 1.2 |
| 10% to 15% F | 9 | 12.6 ± 0.5 | 61.9 ± 5.3 | 2.1 ± 0.2 | 10.1 ± 0.1# | 44.9 ± 0.3# | 11.3 ± 0.2# | 29.1 ± 0.4 | 20.8 ± 0.9# |
| 15% or more F | 5 | 20.8 ± 4.01-153 | 80.4 ± 6.61-155 | 2.4 ± 0.71-154 | 11.6 ± 0.31-153# | 48.0 ± 2.0# | 13.1 ± 0.7# | 25.7 ± 1.6# | 12.6 ± 2.8# |
| Normal control | 11 | NA | NA | NA | 12.8 ± 0.1# | 52.9 ± 0.9# | 14.8 ± 0.2# | 18.6 ± 0.3# | 3.3 ± 0.1# |
Values represent the means ± SEMs.
Hb indicates hemoglobin; MCV, mean corpuscular volume; MCH, mean corpuscular hemoglobin; RDW, red cell distribution width; Retic, Reticulocyte; and NA, not applicable.
Amount of mα2,hγ2 present in the peripheral blood as a percentage of the total Hb.
% 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 10% to 15% F group withP at least < .002.
Nearly statistically significant difference from 10% to 15% group with P = .054.
Not statistically different from 10% to 15% group withP = .59.
#Statistically significant difference from mock group withP at least < .01.