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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.