Figure 4.

ASO-ψ rescues chloride current and splicing for at least 16 days. (A) Average Ieq traces of hBE cells from a homozygous CFTR c.3718-2477C>T patient (donor 3) were treated with ASO-ψ or ASO-C for 4, 8 and 16 days and analyzed for chloride secretion. (B) The area under the curve was calculated after forskolin addition and data was normalized to vehicle-treated cells (±SEM; N = 3; two-way ANOVA with Tukey's multiple comparisons test, **P< 0.01, ****P< 0.0001). (C) RT-PCR analysis of CFTR splicing from RNA isolated from the cells after functional analysis and puromycin treatment. β-Actin is a control for total RNA level. RNA was quantified as percent ψ-Ex splicing and shown below each lane. (D) Quantification of WT-CFTR relative to β-actin from panel (C).