Correction of hematologic pathology and urine concentration defect in mice transplanted with lenti/βAS3 transduced HSCs
Mice . | RBC count, × 106/μL . | Hb level, g/L . | PCV, % . | Reticulocytes, % . | MCV, fL . | MCH, pg . | Urine concentration, mOsm . |
|---|---|---|---|---|---|---|---|
| C57B1/6 controls, n = 5 | 9.0 ± 0.2 | 119 ± 5 | 39.6 ± 1.0 | 4.1 ± 0.7 | 46.1 ± 3.2 | 15.8 ± 1.3 | 1879 ± 231 |
| Sickle controls, n = 5 | 3.3 ± 0.3 | 30 ± 2 | 18.2 ± 2.2 | 69.3 ± 8.6 | 53.7 ± 2.8 | 12.8 ± 1.1 | 849 ± 49 |
| Mock transduced, n = 5 | 2.4 ± 0.3 | 27 ± 4 | 14.0 ± 1.9 | 68.3 ± 3.4 | 54.4 ± 4.6 | 11.3 ± 1.0 | 777 ± 104 |
| Lenti/βAS3 transduced, n = 5 | 6.3 ± 0.2* | 73 ± 7† | 30.0 ± 2.3* | 19.8 ± 2.8* | 46.9 ± 2.3‡ | 11.5 ± 1.1 | 1633 ± 183§ |
| Secondary transplants, n = 5 | 6.1 ± 0.2* | 70 ± 3* | 26.2 ± 0.9* | 16.1 ± 2.0* | 44.6 ± 1.4∥ | 11.5 ± 0.1 | 1922 ± 129* |
Mice . | RBC count, × 106/μL . | Hb level, g/L . | PCV, % . | Reticulocytes, % . | MCV, fL . | MCH, pg . | Urine concentration, mOsm . |
|---|---|---|---|---|---|---|---|
| C57B1/6 controls, n = 5 | 9.0 ± 0.2 | 119 ± 5 | 39.6 ± 1.0 | 4.1 ± 0.7 | 46.1 ± 3.2 | 15.8 ± 1.3 | 1879 ± 231 |
| Sickle controls, n = 5 | 3.3 ± 0.3 | 30 ± 2 | 18.2 ± 2.2 | 69.3 ± 8.6 | 53.7 ± 2.8 | 12.8 ± 1.1 | 849 ± 49 |
| Mock transduced, n = 5 | 2.4 ± 0.3 | 27 ± 4 | 14.0 ± 1.9 | 68.3 ± 3.4 | 54.4 ± 4.6 | 11.3 ± 1.0 | 777 ± 104 |
| Lenti/βAS3 transduced, n = 5 | 6.3 ± 0.2* | 73 ± 7† | 30.0 ± 2.3* | 19.8 ± 2.8* | 46.9 ± 2.3‡ | 11.5 ± 1.1 | 1633 ± 183§ |
| Secondary transplants, n = 5 | 6.1 ± 0.2* | 70 ± 3* | 26.2 ± 0.9* | 16.1 ± 2.0* | 44.6 ± 1.4∥ | 11.5 ± 0.1 | 1922 ± 129* |
Sickle controls are knockout/transgenic sickle mice that were crossed for 8 generations onto the C57B1/6 background. Lenti/βAS3 primary transplants were analyzed 5 (n = 2) or 7 (n = 3) months after transplantation. Secondary transplants are derived from primary transplants number 4 (n = 2) and 5 (n = 3). All secondary transplants were analyzed 7 months after transplantation. Values represent the mean ± SEM. Statistical significance is measured for primary and secondary transplants compared with mock-transduced controls.
Significance was determined by 2-tailed Student t test assuming unequal variances.
Hb indicates hemoglobin; PCV, packed cell volume or hematocrit; MCV, mean corpuscular volume; fL, femtoliter; and MCH, mean corpuscular hemoglobin.
Statistically significant difference with P < .001.
Statistically significant difference with P < .003.
Not statistically different with P = .32.
Statistically significant difference with P < .01.
Not statistically different with P = .13.