Table 2.
Toxicology assessments of mice injected with a single, intramuscular injection of GNE-lipoplex.
| Uninjected (n = 12) | 0 μg (n = 12) | 10 μg (n = 12) | 40 μg (n = 12) | |
|---|---|---|---|---|
| Survival | 12/12 | 12/12 | 12/12 | 12/12 |
| 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 | 0/12 | 0/12 |
| 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 | Uninjected (n = 4) | 0 μg (n = 5) | 10 μg (n = 2) | 40 μg (n = 5) |
| WBC (×103/μL) | 3.44 ± 1.47 | 2.58 ± 0.88 | 2.35 | 4.38 ± 1.5 |
| RBC (×106/μL) | 8.44 ± 0.65 | 7.79 ± 0.73 | 8.81 | 8.43 ± 0.75 |
| Platelets (×103/μL) | 520.75 ± 374.78 | 396.2 ± 343.05 | 569.50 | 428.2 ± 346.31 |
| Serum chemistry | Uninjected (n = 6) | 0 μg (n = 6) | 10 μg (n = 6) | 40 μg (n = 6) |
| AST (U/L) | 172 ± 42 | 305 ± 65* | 323 ± 271 | 141 ± 88 |
| ALT (U/L) | 50 ± 11 | 61 ± 12 | 58 ± 18 | 41 ± 17 |
| Total bilirubin (mg/dL) | 0.1 | 0.1 | 0.1 | 0.1 |
| Alkaline phosphatase (U/L) | 94 ± 17 | 95 ± 17 | 97 ± 13 | 96 ± 18 |
| Creatine kinase (U/L) | 1052 ± 290 | 2110 ± 571* | 2349 ± 2507 | 948 ± 851 |
| BUN (mg/dL) | 20 ± 4 | 22 ± 3 | 24 ± 9 | 17 ± 4 |
| Albumin (g/dL) | 3.1 ± 0.2 | 3.1 ± 0.2 | 3.4 ± 0.2 | 3.1 ± 0.2 |
*
p< 0.05 compared to uninjected cohort.