Table 3.
Toxicology assessments of mice injected with a single, intravenous injection of GNE-lipoplex.
| 0 μg (n = 12) | 10 μg (n = 12) | 40 μg (n = 12) | 100 μg (n = 12) | |
|---|---|---|---|---|
| Survival | 12/12 | 12/12 | 12/12 | 8/12 |
| Treatment induced body weight decrease | 0/12 | 0/12 | 0/12 | 0/12 |
| Acute toxicity (ruffled coat, hunched posture), mice recovered in 48 hours after injection | 0/12 | 0/12 | 2/12 | 8/12 |
| Lesions at necropsy | 0/12 | 0/12 | 0/12 | 0/12 |
| Lesions observed by histopathology caused by treatment | 0/12 | 0/12 | 0/12 | 0/12 |
| Bone marrow function | 0 μg (n = 6) | 10 μg (n = 4) | 40 μg (n = 4) | 100 μg (n = 3) |
| WBC (×103/μL) | 4.13 ± 0.65 | 5.2 ± 0.73 | 4.56 ± 1.09 | 2.7 ± 0.1 |
| RBC (×106/μL) | 8.65 ± 0.65 | 8.61 ± 0.76 | 8.92 ± 0.11 | 8.48 ± 0.45 |
| Platelets (×103/μL) | 623.33 ± 430.04 | 970 ± 378 | 882.75 ± 270.11 | 792 ± 374.2 |
| Serum chemistry | 0 μg (n = 6) | 10 μg (n = 6) | 40 μg (n = 6) | 100 μg (n = 4) |
| AST (U/L) | 102 ± 19 | 144 ± 52 | 119 ± 28 | 88 ± 30 |
| ALT (U/L) | 48 ± 11 | 45 ± 12 | 39 ± 14 | 39 ± 4 |
| Total bilirubin (mg/dL) | 0.1 | 0.1 | 0.1 | 0.1 |
| Alkaline phosphatase (U/L) | 101 ± 14 | 104 ± 24 | 100 ± 22 | 101 ± 19 |
| Creatine kinase (U/L) | 455 ± 122 | 776 ± 390 | 542 ± 131 | 387 ± 220 |
| BUN (mg/dL) | 20 ± 5 | 19 ± 4 | 17 ± 3 | 15 ± 3 |
| Albumin (g/dL) | 3.5 ± 0.2 | 3.3 ± 0.3 | 3.2 ± 0.2 | 3.2 ± 0.2 |