Table 2.
Vector copy number estimation in induced pluripotent stem cells
| Samplea | Provirus | MOI | Cell type | CYBB VCNb | PURO VCNb |
|---|---|---|---|---|---|
| EPN1 | pAlpha.SIN.EFS.PURO.T2A.NGFR.oPRE | 1 | XCGD-iPSC | 0.00 | 1.08 |
| E91PN1 | pAlpha.SIN.EFS.gp91s.F2A.PURO.T2A.NGFR.oPRE | 1 | XCGD-iPSC | 1.10 | 1.07 |
| SPN1 | pAlpha.SIN.SFFV.PURO.T2A.NGFR.oPRE | 1 | Healthy-iPSC | 0.00 | 7.81 |
| S91PN1 | pAlpha.SIN.SFFV.gp91s.F2A.PURO.T2A.NGFR.oPRE | 1 | Healthy-iPSC | 6.75 | 6.33 |
| E91PN10 | pAlpha.SIN.EFS.gp91s.F2A.PURO.T2A.NGFR.oPRE | 10 | XCGD-iPSC | 1.12 | 1.13 |
| S91P10 | pAlpha.SIN.SFFV.gp91s.IRES.PURO.oPRE | 10 | XCGD-iPSC | 14.15 | 13.75 |
CYBB, cytochrome b558 subunit β; gp91s, synthetic gp91 sequence; iPSC, induced pluripotent stem cell; MOI, multiplicity of infection; NGFR, nerve growth factor receptor; PURO, puromycin; VCN, vector copy number (copies/cell); XCGD, X-linked chronic granulomatous disease.
a
For sample names, each letter represents the following: E, EFS; P, PURO; N, NGFR; 91, gp91s.
b
VCN was calculated by the formula: CYBB or PURO copies/RPP30 copies × 2.