Table 1.
Summary of outcomes from high-dose ERT studies conducted on animal models of lysosomal diseases with neurodegeneration.
| Animal model | Immuno- competent |
Doses(n) | Enzyme activity achieved (% Normal) |
Brain storage reduced (%Affected) |
Reduced Neuropathology |
Reference |
|---|---|---|---|---|---|---|
| α-mannosidosis mice | Y | 250 U/kg(2) | ND | 74 % | ND | [9] |
| α-mannosidosis mice | Y | 500 U/kg (2) | 15% | 50% | Y | [10] |
| α-mannosidosis pigs | Y | 10 mg/kg (1) | ND | ND | Y | [14] |
| ASA mice | Y | 20 mg/kg (4) | ND | 30% | Y | [11] |
| ASA mice | N | 50 mg/kg (52) | ND | 34% | Y | [12] |
| AGU mice | Y | 10 mg/kg (8) | 10% | 20% | Y | [6] |
| Krabbe mice | Y | 6 mg/kg | 7% | 18% | Y | [7] |
| MPS II mice | Y | 10 mg/kg (45) | 5% | ND | Y | [13] |
| MPS IIIA mice | Y | 20 mg/kg (4) | 22% | 0 | N | [15] |
| MPS VII mice | N | 20 mg/kg (4) | 2.5% | ND | Y | [8] |
| MPS I mice | Y | 20 mg/kg (4) | 97% | 63% | Y | This study |
Y= yes; N=no; ND = not determined.