Fig. 5: Effect of the lead ASO cocktail on CFTR-W1282X protein expression.
From: Gene-specific nonsense-mediated mRNA decay targeting for cystic fibrosis therapy
a Average traces (n = 3) from Ussing-chamber assay of 16HBE-W1282X cells treated with various combinations of 20 μM ASOs (Sc15, Sc18, LC15-2, or LC18), CFTR correctors (3 μM VX-809 or 3/3 μM VX-661/445), and G418 (200 or 600 μM), as indicated above each trace. All VX-809, VX-661/445, and G418 treatments were started 24 h prior to the assay. Black arrowheads show when Fsk, VX-770, or CFTR inhibitor 172 was added. b Total AUC in (a) divided by the total duration of each experiment (i.e., the time between forskolin treatment and CFTR inhibitor 172 treatment). Abbreviations as in Figs. 1 and 4. Data are represented as mean values ± SD. All data points represent independent biological replicates. n = 3 for all treatments. n.s. P > 0.05, *P < 0.05, **P < 0.01, ***P < 0.001, two-tailed Student’s t-test. Source data are provided as a Source Data File.
