Fig. 2

PDEP-mediated delivery of splice correcting PNA. a Luciferase expression. HeLa pTRE-LucIVS2-705 cells were incubated with varying concentrations of PDEP-705 in serum-free media for 30 min and then incubated for 24 h in complete media. Cell lysates were analyzed for luminescence and standardized by total protein content. The ratios of the RLU μg⁻¹ protein for treated samples divided by untreated samples are plotted. To examine the degree of endosomal entrapment, cells were co-incubated with 120 μM of the endosomolytic agent chloroquine. b mRNA correction. RNA was purified from lysates of cells treated as above and analyzed by qRT-PCR to determine the fold increase in correctly spliced luciferase mRNA standardized to ACTB1. The relative quantification of correctly spliced mRNA over untreated cells was calculated using the Pfaffl model⁵⁶. Error bars are standard deviations of the data, which are shown as open circles, with 3–6 data points, from independent experiments, per average