Table 1.

Animals treated with in vivo FV vector gene therapy

IDWeight at injection, kgAge at injection, dFVDose of vector, IUMobilizationWBCs, × 109/LEstimated MOI*
R2202 NA EF1α.GFP.2A.γC 4.2 × 108 No NA NA 
R2203 NA EF1α.GFP.2A.γC 4.2 × 108 No NA NA 
R2258 1.04 18 EF1α.GFP.2A.γC 4.0 × 108 No 7.67 1.21 
PGK.mCherry.2A.γC 4.0 × 108 
R2260 1.05 18 EF1α.mCherry.2A.γC 4.0 × 108 No 6.64 1.40 
PGK.GFP.2A.γC 4.0 × 108 
H864 0.8 16 PGK.mCherry.2A.γC 4.0 × 108 G-CSF/ AMD3100 16.42 0.28 
H867 1.1 16 PGK.mCherry.2A.γC 4.0 × 108 G-CSF/ AMD3100 18.04 0.26 
IDWeight at injection, kgAge at injection, dFVDose of vector, IUMobilizationWBCs, × 109/LEstimated MOI*
R2202 NA EF1α.GFP.2A.γC 4.2 × 108 No NA NA 
R2203 NA EF1α.GFP.2A.γC 4.2 × 108 No NA NA 
R2258 1.04 18 EF1α.GFP.2A.γC 4.0 × 108 No 7.67 1.21 
PGK.mCherry.2A.γC 4.0 × 108 
R2260 1.05 18 EF1α.mCherry.2A.γC 4.0 × 108 No 6.64 1.40 
PGK.GFP.2A.γC 4.0 × 108 
H864 0.8 16 PGK.mCherry.2A.γC 4.0 × 108 G-CSF/ AMD3100 16.42 0.28 
H867 1.1 16 PGK.mCherry.2A.γC 4.0 × 108 G-CSF/ AMD3100 18.04 0.26 

IU, infectious units; MOI, multiplicity of infection; NA, not available; WBC, white blood cell.

*

MOI at the time of injection was calculated based on the number of virus particles injected divided by the estimated number of circulating WBCs (considering an average blood volume of 86 mL/kg).

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