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Table 1 Study protocol

From: Effect of systemic high dose enzyme replacement therapy on the improvement of CNS defects in a mouse model of mucopolysaccharidosis type II

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
  1. KO knockout, WT wild type, mo month(s)