Table 1.
Summary of targeting locus via CRISPR/Cas9
| Target locus | Protein size (aa) | hPSC line | Donor | Colony number | Heterozygous | Homozygous | Targeting efficiency (%) | Homozygous efficiency (%) |
|---|---|---|---|---|---|---|---|---|
| AAVS1 | 236 | H9 | PC-mNG-SMASh | 7 | 7 | 100 | NT | |
| SOX2 | 317 | H9 | SMASh-Puro SMASh-BSD | 42 | 4 | 35 | 92.9 | 83.3 |
| H1 | SMASh-Puro | 21 | 10 | 8 | 85.7 | 38.1 | ||
| iPSC | SMASh-Puro | 35 | 22 | 5 | 77.1 | 14.3 | ||
| FOXG1 | 489 | H9 | SMASh-Puro | 38 | 6 | 6 | 31.6 | 15.8 |
| SOX17 | 414 | H9 | SMASh-Puro | 39 | 1 | 2 | 7.70 | 5.13 |
| ZNHIT1 | 154 | H9 | SMASh-Puro SMASh-BSD | 36 | 13 | 15 | 77.8 | 41.7 |
| TP53 | 393 | H9 | SMASh-Puro | 41 | 18 | 7 | 61.0 | 17.1 |
| B2M | 119 | H9 | SMASh-Puro SMASh-BSD | 32 | 6 | 21 | 87.5 | 65.6 |
| AAVS1 | 1423 | H9 | PC-Cas9-SMASh | 9 | 6 | 66.7 | NT | |