Table 1.
On-target and off-target cutting analysis of Prkdc ZFN in scid fibroblasts (transduced with IPLV-SFFV-ZFN, qPCR MOI 125) and HSPC (transduced with IPLV-SFFV-ZFN, qPCR MOI 2,500).
| Site | Sample | %Indels mock | %Indels ZFN | %Differential (ZFN-mock) |
|---|---|---|---|---|
| On-target | Fibroblasts | 0.05 | 18.02 | 17.97 |
| Off-target 1 | Fibroblasts | 0.66 | 0.73 | 0.07 |
| Off-target 2 | Fibroblasts | 0.10 | 0.21 | 0.11 |
| Off-target 3 | Fibroblasts | 0.02 | 0.11 | 0.09 |
| Off-target 4 | Fibroblasts | 0.11 | 0.10 | −0.01 |
| Off-target 5 | Fibroblasts | 0.06 | 0.14 | 0.08 |
| Off-target 6 | Fibroblasts | 0.76 | 0.98 | 0.22 |
| Off-target 7 | Fibroblasts | 0.00 | 0.01 | 0.01 |
| Off-target 8 | Fibroblasts | 0.52 | 1.03 | 0.51 |
| Off-target 9 | Fibroblasts | 0.01 | 1.35 | 1.34 |
| On-target | HSPCs | 0.08 | 0.60 | 0.52 |
| Off-target 1 | HSPCs | 0.08 | 0.53 | 0.45 |
| Off-target 2 | HSPCs | 0.08 | 0.16 | 0.08 |
| Off-target 3 | HSPCs | 0.00 | 0.00 | 0.00 |
| Off-target 4 | HSPCs | 0.02 | 0.03 | 0.01 |
| Off-target 5 | HSPCs | 0.04 | 0.03 | −0.01 |
| Off-target 6 | HSPCs | 0.69 | 0.70 | 0.01 |
| Off-target 7 | HSPCs | 0.01 | ND | ND |
| Off-target 8 | HSPCs | 0.29 | 0.18 | −0.11 |
| Off-target 9 | HSPCs | 0.00 | ND | ND |