Table 1 Study protocol
Group (start age) | Subgroup | Dose (mg/kg) | Weekly infusion | Duration (mo) | Assessment | Age (mo) |
|---|---|---|---|---|---|---|
A (2 mo) | n = 7 | 10 | 4 times | 1 | Tissue GAGs | 3 |
Brain pathology | 3 | |||||
n = 7 | 5 | |||||
n = 7 | 1 | |||||
Footprint analysis | 3 | |||||
n = 7 | 1 + mannitol | |||||
KO | n = 5 | Saline | ||||
WT | n = 5 | Saline | ||||
B (2 mo) | n = 7 | 10 | 24 times | 6 | Tissue GAGs | 8 |
Brain pathology | 8 | |||||
n = 7 | 5 | |||||
n = 7 | 5 + mannitol | Footprint analysis | 8 | |||
n = 7 | 10 (2mo) → 5 (4mo) | |||||
n = 7 | 10 (2mo) → 2 (4mo) | Y-maze analysis | 8 | |||
KO | n = 5 | Saline | ||||
WT | n = 5 | Saline | ||||
C (8 mo) | n = 7 | 10 | 12 times | 3 | Tissue GAGs | 11 |
n = 7 | 5 | Brain pathology | 11 | |||
n = 7 | 1 | Footprint analysis | 11 | |||
KO | n = 5 | Saline | Y-maze analysis | 11 | ||
WT | n = 5 | Saline |