Table 5.
AAV Copy Number in Tissues
| Group Assignment | AAV Copy Number in Tissues (100 vg/ng DNA) |
|||
|---|---|---|---|---|
| Brain | Liver | Heart | Spleen | |
| MPS I, untreated (n = 4) | ND | ND | ND | ND |
| Normal (n = 4) | ND | ND | ND | ND |
| MPS I, low dose (n = 6) | ND | 0.09 ± 0.01 | 0.29 ± 0.11 | ND |
| MPS I, middle dose (n = 5) | 0.07 ± 0.03 | 2.0 ± 0.78 | 1.0 ± 0.21 | ND |
| MPS I, high dose (n = 5) | 1.0 ± 0.27 | 51.2 ± 20.7 | 151.2 ± 53.1 | 0.07 ± 0.06 |
| MPS I, high dose, 1 month after dosing (n = 3) | N/A | 135.1 ± 64.6 | N/A | N/A |
Data are shown as mean ± SEM. ND, non-detectable; N/A, not available.