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Table 2 Gb3 levels in mouse tissues after tail vein administration of rAAV2/8-hAGA vector

From: Characterization of Fabry mice treated with recombinant adeno-associated virus 2/8-mediated gene transfer

Mice group Weeks after injection Gb3 levels (nmol/mg protein)
   Liver Kidney Spleen Heart Brain
Untreated Fabry
mice
  2.498 ± 0.261 7.466 ± 0.743 20.665 ± 5.999 7.179 ± 1.939 2.079 ± 1.099
Treated mice
2 × 1011a
6 0.001 ± 0.001 0.032 ± 0.018 0.014 ± 0.007 0.003 ± 0.001 0.139 ± 0.022
  12 0.010 ± 0.001 0.832 ± 0.108 1.098 ± 0.129 0.058 ± 0.047 0.520 ± 0.192
  24 0.015 ± 0.001 1.948 ± 1.487 1.392 ± 0.215 0.054 ± 0.040 1.832 ± 0.299
Treated mice
2 × 1012b
6 0.005 ± 0.002 0.019 ± 0.007 0.013 ± 0.006 0.002 ± 0.001 0.092 ± 0.043
  12 0.007 ± 0.004 0.640 ± 0.349 0.197 ± 0.054 0.019 ± 0.076 0.325 ± 0.146
  24 0.019 ± 0.020 0.359 ± 0.011 0.695 ± 0.252 0.040 ± 0.019 1.267 ± 0.210
  48 0.046 ± 0.001 0.008 ± 0.001 0.732 ± 0.026 0.069 ± 0.108 1.434 ± 0.097
  60 0.092 ± 0.001 0.023 ± 0.071 0.969 ± 0.049 0.502 ± 0.901 1.640 ± 0.127
Treated mice
ERTc
6 0.002 ± 0.001 0.263 ± 0.062 0.015 ± 0.005 0.003 ± 0.001 0.098 ± 0.025
Wild-type mice   0.036 ± 0.013 0.150 ± 0.013 0.252 ± 0.058 0.036 ± 0.0087 0.025 ± 0.011
  1. Data present as average ± SD, a mice treated with 2 × 1011: rAAV2/8-hAGA (2 × 1011 particles/mouse), b mice treated with 2 × 1012: rAAV2/8-hAGA (2 × 1012 particles/mouse), n = 3. cERT treated mice: 1.0 mg/kg once a week for 6 consecutive weeks.