Table 3.
hLDLR mRNA expression in NHP liver
| Necropsy time point | ID (genotype) | hLDLR message/μg RNA | Average vector GC/μg DNA |
|---|---|---|---|
| Day 28 | 19499 (LDLR+/−) | 5.78 × 104 | 3.21 × 106 |
| 19269 (LDLR+/−) | 3.52 × 105 | 2.37 × 107 | |
| Day 365 | 19498 (LDLR+/−) | 5.24 × 104 | 2.37 × 106 |
| 3.30 × 104 | |||
| 20031 (LDLR+/−) | 4.12 × 104 | 5.12 × 106 | |
| 3.67 × 104 | |||
| 090-0263 (wild type) | 3.62 × 104 | 3.47 × 106 | |
| 3.73 × 104 | |||
| 090-0287 (wild type) | 5.67 × 104 | 6.41 × 106 | |
| 4.99 × 104 |
Wild-type and LDLR+/− rhesus macaques were injected i.v. with 1.25 × 1013 GC/kg AAV8.TBG.hLDLR. Following tissue harvest at necropsy, RNA was extracted from the liver, and mRNA levels for the hLDLR transgene were determined.