Table 1.
α-Gal A activity in the organs of wild-type, noninjected α-gal A knockout mice, and α-gal A knockout mice injected with rAAV-CAG-hAGA
| Weeks | Group of mice | Enzyme activity, nmol per h per mg of protein
|
|||||
|---|---|---|---|---|---|---|---|
| Liver | Kidney | Heart | Spleen | Lung | SI | ||
| 4 | Wild type | 24.5 ± 3.1 | 6.5 ± 0.3 | 1.0 ± 0.7 | 53.6 ± 28 | 13.6 ± 2.3 | 33.8 ± 1.1 |
| Noninjected Fabry | 1.1 ± 0.1 | 0.7 ± 0.0 | 0.2 ± 0.0 | 3.5 ± 1.1 | 0.7 ± 0.2 | 1.1 ± 0.1 | |
| Injected Fabry | 109.0 ± 40.0* | 2.0 ± 0.4* | 3.5 ± 0.8* | 34.1 ± 6.6† | 1.3 ± 0.2* | 3.5 ± 0.7* | |
| 8 | Wild type | 24.1 ± 4.6 | 6.2 ± 0.4 | 1.3 ± 0.1 | 47.2 ± 11 | 12.4 ± 1.1 | 21.1 ± 5.0 |
| Noninjected Fabry | 1.5 ± 0.4 | 0.9 ± 0.2 | 0.2 ± 0.0 | 4.3 ± 0.6 | 0.6 ± 0.1 | 1.1 ± 0.1 | |
| Injected Fabry | 183.0 ± 88.0* | 3.9 ± 1.7* | 3.3 ± 1.1† | 80.3 ± 42* | 1.9 ± 0.5* | 6.7 ± 2.7* | |
| 12 | Wild type | 22.2 ± 4.9 | 6.5 ± 0.1 | 1.4 ± 0.1 | 37.5 ± 5.6 | 13.5 ± 2.9 | 18.2 ± 0.8 |
| Noninjected Fabry | 1.1 ± 0.1 | 0.8 ± 0.1 | 0.2 ± 0.6 | 3.7 ± 0.7 | 0.8 ± 0.4 | 1.0 ± 0.2 | |
| Injected Fabry | 301.0 ± 285.0 | 3.8 ± 1.8 | 4.2 ± 1.9* | 82.7 ± 47.0 | 2.7 ± 1.1* | 7.5 ± 1.0 | |
| 24 | Wild type | 20.5 ± 6.3 | 8.8 ± 0.7 | 1.6 ± 0.2 | 38.1 ± 6.7 | 20.6 ± 8.6 | 27.1 ± 4.9 |
| Noninjected Fabry | 2.2 ± 1.1 | 0.9 ± 0.2 | 0.1 ± 0.0 | 3.6 ± 0.1 | 0.8 ± 0.4 | 0.7 ± 0.1 | |
| Injected Fabry | 113.0 ± 16.0† | 6.2 ± 1.2† | 5.2 ± 0.8† | 68.7 ± 13.0† | 2.0 ± 0.3† | 4.5 ± 2.1* | |
Time in weeks after injection of the vector is shown in the left column. Mean α-gal A activity ± SEM is shown for four mice at each time point. SI, small intestine.
*
P < 0.05 vs. noninjected Fabry mice.
†
P < 0.01.