Table 2.
Editing efficiency in patients’ cells.
| Case | Patient Code | Sample | Total reads | HDR efficiency (%) | In/dels frequency (%) | C (WT) | T (M) |
|---|---|---|---|---|---|---|---|
| 1 | 2204 | Fibroblasts | 22141 | 64.2 | 0.1 | 83% (18284) | 17% (3857) |
| 2 | 304 | Fibroblasts | 2675 | 50 | 0.2 | 68.3% (1833) | 31.5% (842) |
| 3 | 2271 | Fibroblasts | 104 | 83.6 | 0 | 98% (102) | 2% (2) |
| 3 | 2271 | iPS-derived Neurons | 2935 | 14 | 0 | 56% (1643) | 44% (1292) |
| 4 | 2239 | Fibroblasts | 1864 | 20.4 | 0.5 | 59.8% (1120) | 39.7% (744) |
Sample: type of cell analysed; Total reads: number of total reads analysed by NGS; HDR efficiency: percentage of Homology Directed Repair.
Indel frequency: percentage of insertions and deletions caused by Cas9 activity; C (WT): wild type nucleotide; T (M): mutated nucleotide