Figure 9.
Functional analysis of W1282X-CFTR rescue with the HS-YFP assay. (A) Scatter dot plot reporting W1282X-CFTR activity (quenching rate, QR) in cells treated with the indicated combinations of compounds: SMG1i (1 µM), VX-809 (1 µM), VX-445 (5 µM), G418 (0.5 mg/mL). ***, p < 0.001 vs. control. ###, p < 0.001 vs. SMG1i alone. (B) Dose–response relationship for SMG1i in the absence and in the presence of CFTR correctors. ***, p < 0.001 vs. no SMG1i. #, p < 0.05 between indicated conditions. The inset shows representative traces from the HS-YFP assay in which iodide-rich solution is added (arrow) to start fluorescence quenching caused by iodide influx through CFTR. Conditions were vehicle alone (control), 1 µM SMG1i, and the combination of SMG1i plus VX-809 and VX-445 (combo).