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. 2020 Sep 4;8:536. doi: 10.3389/fped.2020.00536

Figure 5.

Figure 5

CFTR function measurements in EpiXTM-expanded CF patient cultures. CF nasal epithelial cultures, five CF subjects with different genotypes. (A–E) Top. Detailed recordings of CFTR currents. Traces were from cultures treated with CFTR corrector VX-809. Chloride currents (ICl) were recorded in presence of amiloride. Note differences in scale, reflective of decreasing CFTR function with CF genotypes. CFTRinh refers to sequential addition of three CFTR inhibitor compounds (CFTRinh-172, PPQ-102, and GlyH-101). Middle (F–J): Individual responses to forskolin (baseline) and forskolin plus VX-770 (VX-770). Baseline represents change in cAMP-dependent CFTR current by forskolin alone. VX-770 represents change in current by forskolin+VX-770. *denotes significant increase by VX-770 (paired t-test, p < 0.05). Bottom (K–O): Average stimulated and blocked CFTR currents of non-treated cultures (no corr) and cultures treated with CFTR corrector VX-809 or VX-661. Upward bars present chloride current change (ΔICl) after stimulation with forskolin alone (cAMP; gray) or after stimulation with forskolin plus VX-770 (white, VX-770). Downward bars present ΔICl after maximal block by CFTR inhibitors (CFTRinh; black). Bars represent mean ± SE. *, significantly different by unpaired t-test (p < 0.05). (A,F,K) F508del/R117H-7T, passage 3 (PD = 8.7). Non-treated: n = 5; VX-809 treated: n = 4; VX-661 treated: n = 4. (B,G,L) R334W/406-1G>A, passage 3 (PD = 8.7). Non-treated: n = 3; VX-809 treated: n = 4; VX-661 treated: n = 3. (C,H,M) F508del/c.850dupA, passage 4 (PD = 11.6). Non-treated: n = 2; VX-809 treated: n = 3; VX-661 treated: n = 3. (D,I,N) F508del homozygote, passage 3 (PD = 10.4). Non-treated: n = 5; VX-809 treated: n = 4; VX-661 treated: n = 4. (E,J,O) CFTRdele2,3(21 kb) homozygote, passage 3 (PD = 10.4). Non-treated: n = 4; VX-809 treated: n = 2; VX-661 treated: n = 3.