Table 3.
Tissue GAG levels of MPS I mice treated with different doses and volumes of lentiviral vector. Normal heterozygous mice had 4.3 ± 0.4 μg GAG/mg protein in the brain, 1.3 ± 1.3 in the heart, 7.4 ± 3.4 in the liver and 3.6 ± 1.0 in the spleen. ⁎The difference between treated mice and untreated MPS I mice was significant, with a p value of < 0.05.
| μg GAG/mg protein |
|||||
|---|---|---|---|---|---|
| Volume (μL) | Brain | Heart | Liver | Spleen | |
| CPGKID (high dose, n = 4) | 50 | 12.2 ± 0.39 | 7.4 ± 0.84⁎ | 7.2 ± 1.1⁎ | 13.9 ± 2.8 |
| CPGKID (low dose, n = 8) | 50 | 15.7 ± 2 | 27.1 ± 2.9⁎ | 9.6 ± 4.1 | 11.1 ± 1.8 |
| CPGKID (high dose, n = 5) | 100 | 11.3 ± 0.47 | 6.9 ± 1.3⁎ | 6.5 ± 0.5⁎ | 8 ± 1.1 |
| Untreated MPS I (n = 5) | N/A | 12.5 ± 2.3 | 18.1 ± 3.8 | 62.3 ± 16.1 | 18.7 ± 5.7 |