Table 2.

Biodistribution of AAV6-cFVIII vector DNA in hemophilia A mice


Tissue

Naive

M1*

M2*

M3*

M4*

Mean ± SD
Gonad   0.0000   0.0012   0.0007   0.0017   0.0017   0.001 ± 0.000  
Liver   0.0000   4.7044   11.3035   5.2088   1.8963   5.778 ± 1.981  
Lung   0.0000   0.0004   0.0018   0.0001   0.0060   0.002 ± 0.001  
Heart   0.0001   0.0013   0.0042   0.0004   0.0015   0.002 ± 0.001  
Kidney   0.0000   0.0075   0.0064   0.0008   0.0010   0.004 ± 0.002  
Spleen
 
0.0002
 
0.0227
 
0.0374
 
0.0217
 
0.0155
 
0.024 ± 0.005
 

Tissue

Naive

M1*

M2*

M3*

M4*

Mean ± SD
Gonad   0.0000   0.0012   0.0007   0.0017   0.0017   0.001 ± 0.000  
Liver   0.0000   4.7044   11.3035   5.2088   1.8963   5.778 ± 1.981  
Lung   0.0000   0.0004   0.0018   0.0001   0.0060   0.002 ± 0.001  
Heart   0.0001   0.0013   0.0042   0.0004   0.0015   0.002 ± 0.001  
Kidney   0.0000   0.0075   0.0064   0.0008   0.0010   0.004 ± 0.002  
Spleen
 
0.0002
 
0.0227
 
0.0374
 
0.0217
 
0.0155
 
0.024 ± 0.005
 

The copy numbers of canine FVIII DNA per diploid genome as determined by QPCR. The limit of detection is 0.0003 copy/diploid genome.

*

Individual mice dosed with 3 × 1011 vg AAV6-cFVIII.

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