Greater improvement of the β-thalassemic phenotype with the LG/HA-Y1 vector than with the LG vector at low copy number in secondary transplants
| Group . | Copy per WBC . | RBC+ . | hu β-glo . | Hb . | Hc . | RTC . | RBC . | MCV . | MCH . | MChuH . | Sol Hb . | WBC . | ||||||||||||
|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|
| Mean . | SD . | Mean . | SD . | Mean . | SD . | Mean . | SD . | Mean . | SD . | Mean . | SD . | Mean . | SD . | Mean . | SD . | Mean . | SD . | Mean . | SD . | Mean . | SD . | Mean . | SD . | |
| 1 | 0.00 | 0.00 | 0.00 | 0.0 | 0.0 | 0.0 | 11.1 | 0.2 | 34.4 | 1.1 | 22.0 | 2.8 | 9.3 | 0.2 | 37.0 | 1.6 | 11.9 | 0.4 | 0.0 | 0.0 | 9.2 | 0.4 | 7.4 | 1.1 |
| 2 | 0.04 | 0.04 | 17.8 | 24.5 | 5.9 | 7.2 | 11.4 | 0.9 | 35.4 | 2.2 | 17.8 | 4.6 | 9.4 | 0.2 | 37.6 | 1.8 | 12.1 | 0.8 | 4.6 | 0.9 | 9.9 | 1.1 | 8.6 | 2.7 |
| 3 | 0.07 | 0.08 | 75.9 | 16.4 | 26.1 | 5.6 | 14.1 | 1.5 | 44.0 | 5.2 | 6.9 | 5.1 | 10.1 | 1.0 | 43.4 | 1.5 | 13.9 | 0.4 | 4.8 | 0.5 | 13.2 | 1.9 | 8.4 | 1.0 |
| P | .279 | .001 | < .001 | .011 | .011 | .004 | .207 | .001 | .003 | .009 | .008 | .694 | ||||||||||||
| S1-2 | N | N | N | N | N | N | N | Y | Y | |||||||||||||||
| S1-3 | Y | Y | Y | Y | Y | Y | Y | Y | Y | |||||||||||||||
| S2-3 | Y | Y | Y | Y | Y | Y | Y | N | N | |||||||||||||||
| Group . | Copy per WBC . | RBC+ . | hu β-glo . | Hb . | Hc . | RTC . | RBC . | MCV . | MCH . | MChuH . | Sol Hb . | WBC . | ||||||||||||
|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|
| Mean . | SD . | Mean . | SD . | Mean . | SD . | Mean . | SD . | Mean . | SD . | Mean . | SD . | Mean . | SD . | Mean . | SD . | Mean . | SD . | Mean . | SD . | Mean . | SD . | Mean . | SD . | |
| 1 | 0.00 | 0.00 | 0.00 | 0.0 | 0.0 | 0.0 | 11.1 | 0.2 | 34.4 | 1.1 | 22.0 | 2.8 | 9.3 | 0.2 | 37.0 | 1.6 | 11.9 | 0.4 | 0.0 | 0.0 | 9.2 | 0.4 | 7.4 | 1.1 |
| 2 | 0.04 | 0.04 | 17.8 | 24.5 | 5.9 | 7.2 | 11.4 | 0.9 | 35.4 | 2.2 | 17.8 | 4.6 | 9.4 | 0.2 | 37.6 | 1.8 | 12.1 | 0.8 | 4.6 | 0.9 | 9.9 | 1.1 | 8.6 | 2.7 |
| 3 | 0.07 | 0.08 | 75.9 | 16.4 | 26.1 | 5.6 | 14.1 | 1.5 | 44.0 | 5.2 | 6.9 | 5.1 | 10.1 | 1.0 | 43.4 | 1.5 | 13.9 | 0.4 | 4.8 | 0.5 | 13.2 | 1.9 | 8.4 | 1.0 |
| P | .279 | .001 | < .001 | .011 | .011 | .004 | .207 | .001 | .003 | .009 | .008 | .694 | ||||||||||||
| S1-2 | N | N | N | N | N | N | N | Y | Y | |||||||||||||||
| S1-3 | Y | Y | Y | Y | Y | Y | Y | Y | Y | |||||||||||||||
| S2-3 | Y | Y | Y | Y | Y | Y | Y | N | N | |||||||||||||||
Mean hematological parameters and SDs in groups of secondary transplanted recipients with mock (1)-, LG (2)-, and LG/HA-Y1 (3)-modified cells 10 weeks after secondary transplantation.
Copy indicates peripheral blood copy number per cell; RBC+, RBC containing human hemoglobin (%); hu β-glo, human β-globin (%); Hb, hemoglobin (g/dL); Hc, hematocrit value (%); RTC, reticulocytes (%); RBC, red blood cells ( × 1012/L); MCV, mean corpuscular volume (fL); MCH, mean corpuscular hemoglobin (pg); MChuH, mean corpuscular human hemoglobin in modified RBC (pg); sol Hb, soluble hemoglobin (g/dL); WBC, white blood cells ( × 109/L).
Multiple and pairwise comparisons are made by the use of one-way analysis of variance and the Holm-Sidak method, respectively. When an overall significance level < .05 (P value) is obtained, statistical significance is given as yes (Y) or no (N) between groups 1 and 2 (S1-2), 1 and 3 (S1-3), and 2 and 3 (S2-3).