Table 2.
IDUA transgene expression in tissues of MPS I mice treated with different doses and volumes of lentiviral vector. High dose: 1 × 107 TU/g; low dose: 5 × 106 TU/g. ND: non-detectable (< 0.002 nmol/h/mg). Normal heterozygous mice had IDUA levels: 2.7 ± 0.26 nmol/h/mg protein in the brain, 3.8 ± 0.06 in the heart, 1.8 ± 0.1 in the liver and 10.2 ± 0.23 in the spleen. Data are mean ± standard errors. ⁎The difference between treated mice and untreated MPS I mice was significant, with a p value of < 0.05.
| IDUA activity (nmol/h/mg protein) |
|||||
|---|---|---|---|---|---|
| Volume (μL) | Brain | Heart | Liver | Spleen | |
| CPGKID (high dose, n = 4) | 50 | ND | 0.09 ± 0.07 | 2 ± 0.54⁎ | 9.1 ± 3.7⁎ |
| CPGKID (low dose, n = 8) | 50 | 0.006 ± 0.01 | 0.01 ± 0.01 | 0.25 ± 0.05⁎ | 0.41 ± 0.1⁎ |
| CPGKID (high dose, n = 5) | 100 | ND | 0.34 ± 0.13⁎ | 1.7 ± 0.36⁎ | 2.1 ± 0.47⁎ |
| Untreated MPS I (n = 5) | N/A | ND | ND | ND | ND |