Table 3

Reconstitution in spleens of mice following ex vivo bone marrow HSC gene transfer with the A2UCOE-IL2RG and SFFV-IL2RG lentiviral vector

CD3, %CD4, %CD8, %B220/IgM, %IgM, %
Mock 0.46 0.74 1.4 0.55 0.64 
A2UCOE-γ c 1 18.1 22.4 38.5 12.5 
A2UCOE-γ c 2 30.4 25.7 7.4 24.3 22.5 
A2UCOE-γ c 3 34 19.2 7.4 9.5 24 
A2UCOE-γ c 4 37 29 9.3 18.1 22 
A2UCOE-γ c 5 23 17.4 7.7 5.1 9.4 
SFFV-γ c 1 14.7 10.9 3.4 4.5 
SFFV-γ c 2 9.3 8.5 1.5 2.6 1.5 
Control 15.9 10.9 6.2 68.6 6.5 
CD3, %CD4, %CD8, %B220/IgM, %IgM, %
Mock 0.46 0.74 1.4 0.55 0.64 
A2UCOE-γ c 1 18.1 22.4 38.5 12.5 
A2UCOE-γ c 2 30.4 25.7 7.4 24.3 22.5 
A2UCOE-γ c 3 34 19.2 7.4 9.5 24 
A2UCOE-γ c 4 37 29 9.3 18.1 22 
A2UCOE-γ c 5 23 17.4 7.7 5.1 9.4 
SFFV-γ c 1 14.7 10.9 3.4 4.5 
SFFV-γ c 2 9.3 8.5 1.5 2.6 1.5 
Control 15.9 10.9 6.2 68.6 6.5 

SCID-X1 mice were subjected to an ex vivo procedure with HSCs transduced with the A2UCOE-IL2RG lentiviral vector (Figure 1B). At 3 months following engraftment, spleens were excised and analyzed for reconstitution of T-, B-, and NK cell lineages by FACS. Cells were stained with anti-CD8, -CD4, -NK1.1, -IgM, and -B220 antibodies. Efficient reconstitution of all cell lineages is observed in A2UCOE-IL2RG vector and SFFV-IL2RG–positive control transduced mice.

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