Table 3. Tissue GUS levels after GUS infusion into MPS VII/E540ATg mice at different doses and regimens.
| Group | Dose mg/kg | Doses, n | Liver | Spleen | Heart | Kidney | Bone | Bone marrow | Muscle | Eye |
|---|---|---|---|---|---|---|---|---|---|---|
| I | 0 | 3 | 0.35 ± 0.08 | 0.41 ± 0.36 | 0.053 ± 0.030 | 0.04 ± 0.02 | 0.39 ± 0.05 | — | — | 0.019 ± 0.004 |
| 0.2 | 0.1 | 0.25 | 0.03 | 0.39 | — | — | 0.39 | |||
| 0.3 | 3 | 13.29 ± 4.15 | 1.63 ± 0.61 | 0.36 ± 0.12 | 0.39 ± 0.15 | 1.27 ± 0.03 | — | — | 0.074 ± 0.027 | |
| 7.2 | 0.5 | 1.72 | 0.36 | 1.27 | — | — | 1.5 | |||
| 1.0 | 2 | 41.88 ± 14.76 | 4.49 ± 1.63 | 0.83 ± 0.05 | 0.64 ± 0.1 | 3.57 ± 0.56 | — | — | 0.126 ± 0.025 | |
| 22.6 | 1.5 | 4.0 | 0.6 | 3.6 | — | — | 2.6 | |||
| 2.5 | 2 | 96.02 ± 5.44 | 44.38 ± 24.5 | 2.16 ± 0.09 | 3.18 ± 1.18 | 11.78 ± 0.03 | — | — | 0.166 ± 0.049 | |
| 51.9 | 14.7 | 10.4 | 2.9 | 11.8 | — | — | 3.4 | |||
| 5.0 | 3 | 211.8 ± 121 | 63.83 ± 22.3 | 3.36 ± 1.29 | 5.47 ± 2.98 | 8.66 ± 4.75 | — | — | 0.231 ± 0.024 | |
| 114 | 21.2 | 16.2 | 5.1 | 8.7 | — | — | 4.7 | |||
| II | 0 | 0.31 ± 0.02 | 0.096 ± .016 | 0.023 ± 0.023 | 0.06 ± 0.01 | — | 0.044 ± 0.024 | 0.024 ± .010 | 0.056 ± .023 | |
| 0.17 | 0.03 | 0.1 | 0.06 | — | 0.03 | 0.48 | 1.1 | |||
| 1 | 4 | 65.1 ± 28.4 | 19.0 ± 9.7 | 1.10 ± 0.35 | 1.71 ± 1.07 | — | 6.30 ± 2.84 | 0.64 ± 0.38 | 0.19 ± .006 | |
| 35.2 | 6.3 | 5.3 | 1.58 | — | 3.91 | 12.9 | 3.9 | |||
| 2 | 5 | 108 ± 25.9 | 132 ± 105 | 1.78 ± 0.70 | 3.69 ± 1.14 | — | 14.34 ± 9.62 | 0.81 ± 0.43 | 0.28 ± .08 | |
| 58.4 | 43.9 | 8.6 | 3.42 | — | 8.9 | 16.4 | 5.7 | |||
| 4 | 5 | 230 ± 31.3 | 113 ± 52 | 4.18 ± 2.09 | 7.83 ± 1.13 | — | 46.44 ± 20.51 | 2.04 ± 1.56 | 0.60 ± .23 | |
| 124 | 37.5 | 20.1 | 7.25 | — | 28.8 | 41.2 | 12.3 | |||
| IV | 20 | 2 | 975 ± 442 | 374 ± 38 | 11.5 ± 1 | 20 ± 1.9 | — | 173 ± 25 | 2.95 ± 1.16 | 1.26 ± 0.39 |
| 527 | 124 | 55.3 | 18.5 | — | 107 | 60.0 | 25.8 | |||
| WtB6 | — | 4 | 185 ± 11.9 | 301 ± 26.6 | 20.8 ± 12.5 | 108 ± 7.5 | 100 ± 18 | 161 ± 35 | 4.95 ± 1.80 | 4.88 ± 0.68 |
Data are given as means ± SD, with the percentage of wild-type (Wt) values shown in italic type. —, not applicable.