Table 2.
Summary of CFTR measurements in EpiXTM expanded CF nasal cultures.
| Summary of CFTR Function and Modulator Responses 100% WT CFTR = −17.85 ± 2.21 μA/cm2 (n=12) | |||
|---|---|---|---|
| Genotype | Treatment | CFTR current (μA/cm2, mean ± SE) | Normal CFTR (%) |
| F508del/R117H-7T | No corrector | −10.04 ± 0.69 (n = 5) | 56 |
| F508del/R117H-7T | VX-809 | −13.06 ± 0.53 (n = 4)* | 73 |
| F508del/R117H-7T | VX-661 | −12.01 ± 0.47 (n = 4) | 67 |
| F508del/F508del | No corrector | −2.14 ± 0.39 (n = 5) | 12 |
| F508del/F508del | VX-809 | −2.63 ± 0.52 (n = 4) | 15 |
| F508del/F508del | VX-661 | −2.19 ± 0.30 (n = 4) | 12 |
| F508del/c.850dupA | No corrector | −2.07 ± 0.04 (n = 2) | 12 |
| F508del/c.850dupA | VX-809 | −3.21 ± 0.35 (n = 3)* | 18 |
| F508del/c.850dupA | VX-661 | −3.19 ± 1.15 (n = 3) | 18 |
| R334W/406-1G>A | No corrector | −4.20 ± 1.38 (n = 3) | 24 |
| R334W/406-1G>A | VX-809 | −4.81 ± 1.06 (n = 4) | 27 |
| R334W/406-1G>A | VX-661 | −3.43 ± 1.03 (n = 3) | 19 |
| CFTRdele2,3(21 kb)/CFTRdele2,3(21 kb) | No corrector | −1.68 ± 0.32 (n = 4) | 9 |
| CFTRdele2,3(21 kb)/CFTRdele2,3(21 kb) | VX-809 | −1.28 ± 0.05 (n = 2) | 7 |
| CFTRdele2,3(21 kb)/CFTRdele2,3(21 kb) | VX-661 | −3.52 ± 0.05 (n = 3)** | 20 |
All CFTR currents were measured in response to stimulation by forskolin and acute addition of VX-770 in uncorrected, VX-809 corrected or VX-661 corrected cultures. CFTR current refers to blockable current. Significance for CFTR corrector treatment is marked with
*
(p < 0.05) and
**
(p < 0.01), determined by unpaired t-tests.