Table 3. Pre and post infusion VCN of cells transduced with LV X-SCID 2nd gen in the presence or absence of transduction enhancers.
CD34+ cells from two normal donors were transduced with LV X-SCID 2nd gen and transplanted into NBSGW mice (n=5 per group). The mean VCN ± standard deviation (SD) for pre-infused cells, and blood (12 weeks), whole BM, BM hCD34+ cells, and spleen (16 weeks) post transplantation are shown. The fold decrease in VCN of indicated tissues [preinfusion VCN/mean VCN of each group] is shown below. VCN, vector copy number, LV, Lentiviral vector; PGE2, prostaglandin E2; BM, bone marrow.
| VCN | LV | PGE2 | LentiBOOST | LentiBOOST+PGE2 | ||||
|---|---|---|---|---|---|---|---|---|
| Donor 1 | Donor 2 | Donor 1 | Donor 2 | Donor 1 | Donor 2 | Donor 1 | Donor 2 | |
| Preinfusion | 2.30 | 1.06 | 5.60 | 2.44 | 4.63 | 2.77 | 6.68 | 4.42 |
| Blood | 0.64 ± 0.03 | 0.43 ± 0.07 | 1.53 ± 0.13 | 0.80 ± 0.09 | 2.31 ± 0.35 | 1.77 ± 0.23 | 2.54 ± 0.6 | 1.81 ± 0.31 |
| BM | 0.86 ± 0.15 | 0.43 ± 0.04 | 2.14 ± 0.29 | 0.91 ± 0.10 | 3.05 ± 0.79 | 1.89 ± 0.12 | 2.83 ± 0.87 | 2.35 ± 0.91 |
| hCD34+ | 0.89 ± 0.18 | 0.45 ± 0.06 | 2.15 ± 0.42 | 0.88 ± 0.13 | 3.12 ± 0.78 | 1.97 ± 0.39 | 2.95 ± 1.33 | 2.43 ± 0.92 |
| Spleen | 0.64 ± 0.06 | 0.37 ± 0.05 | 1.58 ± 0.18 | 0.70 ± 0.10 | 2.36 ± 0.34 | 1.62 ± 0.17 | 2.58 ± 0.34 | 1.96 ± 0.31 |
| Fold decrease versus preinfusion VCN | ||||||||
| Blood | 3.62 | 2.49 | 3.66 | 3.04 | 1.79 | 1.57 | 2.63 | 2.44 |
| BM | 2.66 | 2.49 | 2.62 | 2.69 | 1.35 | 1.46 | 2.36 | 1.88 |
| hCD34+ | 2.59 | 2.37 | 2.61 | 2.76 | 1.32 | 1.40 | 2.27 | 1.82 |
| Spleen | 3.62 | 2.86 | 3.55 | 3.51 | 1.75 | 1.71 | 2.59 | 2.26 |