UROD Enzymatic Activity and Porphyrin Level in Untransduced and Transduced HEP LB Cells Before and After FACS Selection
| Cell Population . | UROD Activity (U/mg) . | %v LBHEPLUDSN11 . | Porphyrin Level (fmol/1,000 cells) . | % v LBHEP . |
|---|---|---|---|---|
| Experiment no. 1 | ||||
| LB44 | 219 ± 123 | 11 | 9.29 ± 1.92 | 100 |
| LB44-LUDSN11 | 1,927 ± 266 | 100 | 0.29 ± 0.34 | 3 |
| LB44/LB44-LUDSN11 (80/20) | 647 ± 187 | 34 | 7.71 ± 0.09 | 83 |
| FACS sorted | ||||
| High fluorescence | 539 ± 101 | 28 | 7.88 ± 2.24 | 85 |
| Low fluorescence | 1,459 ± 148 | 76 | 0.95 ± 0.13 | 10 |
| Experiment no. 2 | ||||
| LB45 | 288 ± 108 | 10 | 13.28 ± 2.53 | 100 |
| LB45-LUDSN11 | 2,901 ± 543 | 100 | 0.06 ± 0.02 | 0.5 |
| LB45/LB45-LUDSN11 (80/20) | 1,100 ± 308 | 38 | 9.94 ± 0.18 | 75 |
| FACS sorted | ||||
| High fluorescence | 880 ± 236 | 30 | 6.53 ± 1.62 | 49 |
| Low fluorescence | 3,521 ± 1,635 | 121 | 0.64 ± 0.58 | 5 |
| Cell Population . | UROD Activity (U/mg) . | %v LBHEPLUDSN11 . | Porphyrin Level (fmol/1,000 cells) . | % v LBHEP . |
|---|---|---|---|---|
| Experiment no. 1 | ||||
| LB44 | 219 ± 123 | 11 | 9.29 ± 1.92 | 100 |
| LB44-LUDSN11 | 1,927 ± 266 | 100 | 0.29 ± 0.34 | 3 |
| LB44/LB44-LUDSN11 (80/20) | 647 ± 187 | 34 | 7.71 ± 0.09 | 83 |
| FACS sorted | ||||
| High fluorescence | 539 ± 101 | 28 | 7.88 ± 2.24 | 85 |
| Low fluorescence | 1,459 ± 148 | 76 | 0.95 ± 0.13 | 10 |
| Experiment no. 2 | ||||
| LB45 | 288 ± 108 | 10 | 13.28 ± 2.53 | 100 |
| LB45-LUDSN11 | 2,901 ± 543 | 100 | 0.06 ± 0.02 | 0.5 |
| LB45/LB45-LUDSN11 (80/20) | 1,100 ± 308 | 38 | 9.94 ± 0.18 | 75 |
| FACS sorted | ||||
| High fluorescence | 880 ± 236 | 30 | 6.53 ± 1.62 | 49 |
| Low fluorescence | 3,521 ± 1,635 | 121 | 0.64 ± 0.58 | 5 |
The mean activity is shown for three determinations in one experiment for the mixture LB44/LB44-LUDSN11 (80/20) and in three independent experiments for the mixture LB45/LB45-LUDSN11 (80/20). Theoretical values of UROD activity were 561 U/mg for the mixture in experiment no. 1 and 811 U/gm for the mixture in experiment no. 2. Data are the mean ± SD.