Table 1.
Summary of percent editing of CF upper airway basal cells (UABCs) and human bronchial epithelial cell (HBEC) samples and relative restoration of CFTR function as measured by responses to CFTRinh-172
| Patient | Genotype | Editing efficiency (% tCD19+ cells) | Raw-inhibitable CF current μA/cm2 | Percentage of non-CF-inhibitable current |
|---|---|---|---|---|
| UABCs | ||||
| WT average: −19 ± 11 μA/cm2 | ||||
| 1 | ΔF/ΔF | 67.7 | −13.1 | 69.81 |
| 1 | ΔF/ΔF | 67.7 | −12.78 | 68.10 |
| 1 | ΔF/ΔF | 67.7 | −8.96 | 47.75 |
| 1 | ΔF/ΔF | 67.7 | −7.8 | 41.57 |
| 2 | ΔF/3659delC | 56.8 | −9.15 | 48.76 |
| 2 | ΔF/3659delC | 56.8 | −24.8 | 132.16 |
| 2 | ΔF/3659delC | 56.8 | −28.74 | 153.15 |
| 3 | ΔF/ΔF | 68.5 | −8.79 | 46.84 |
| 3 | ΔF/ΔF | 68.5 | −6.14 | 32.72 |
| 4 | ΔF/ΔF | 82.3 | −25.59 | 136.37 |
| 4 | ΔF/ΔF | 82.3 | −10.75 | 57.29 |
| 4 | ΔF/ΔF | 82.3 | −9.04 | 48.17 |
| HBECs | ||||
| WT average: −18 ± 9 μA/cm2 | ||||
| 5 | ΔF/ΔF | 57.0 | −10.9 | 57.31 |
| 5 | ΔF/ΔF | 57.0 | −11.72 | 61.62 |
| 6 | ΔF/ΔF | 79.0 | −22.31 | 117.29 |
| 6 | ΔF/ΔF | 79.0 | −16.99 | 89.32 |
| 6 | ΔF/ΔF | 79.0 | −19.53 | 102.68 |
| 6 | ΔF/ΔF | 79.0 | −17.63 | 92.69 |
| 7 | ΔF/N1303K | 74.8 | −17.6 | 92.53 |
| 7 | ΔF/N1303K | 74.8 | −18.36 | 96.53 |
| 7 | ΔF/N1303K | 74.8 | −20.07 | 105.52 |
| 7 | ΔF/N1303K | 74.8 | −15.14 | 79.60 |
| 7 | ΔF/N1303K | 74.8 | −27.75 | 145.89 |
| 8 | 1717-1G/N1303K | 79.1 | −28.94 | 152.15 |
| 8 | 1717-1G/N1303K | 79.1 | −10.9 | 57.31 |
| 9 | ΔF/W1282X | 79.0 | −11.61 | 61.04 |
| 9 | ΔF/W1282X | 68.0 | −12.04 | 63.30 |
| 9 | ΔF/W1282X | 79.0 | −13.06 | 68.66 |
| 9 | ΔF/W1282X | 68.0 | −15.07 | 79.23 |
| 10 | W1282X/W1282X | 89.2 | −12.75 | 67.03 |
| 10 | W1282X/W1282X | 80.9 | −16.02 | 84.2239664 |
| 11 | W1282X/R553X | 73.1 | −16.1 | 84.6445604 |
CFTR contains 1,480 amino acids. The mutations presented here occur in the first 1/3 or the last 1/3 of the CFTR protein. ΔF refers to the F508del mutation.