Table 2.
Pharmacodynamic study.
| Groupa | Number of mice/group | Dose | Miragen compounds | Route of administration | Scheduleb |
|---|---|---|---|---|---|
| 1 | 8 | 100 μL | PBS | i.v. | Injection at 0 and 48 hours after enrollment |
| 2 | 8 | 1 mg/kg | Control oligonucleotide | i.v. | Injection at 0 and 48 hours after enrollment |
| 3 | 8 | 1 mg/kg | Cobomarsen | i.v. | Injection at 0 and 48 hours after enrollment |
Five- to six-week-old female NSG (NOD.Cg-Prkdcscid Il2rgtm1Wjl/SzJ, strain number 005557) mice were divided in three groups: 1, 2, and 3 containing 8 mice each. All mice were injected intravenously in the tail vein with 100 μL of the vehicle PBS, the second with 100 μL of 1 mg/kg control oligonucleotide in PBS, and the third with 100 μL of 1 mg/kg of cobomarsen in PBS.
For the pharmacodynamic study, all the injections were performed at the beginning of the study, indicated as 0 and at 48 hours after enrollment.