Fig. 3.

Cytosolic penetration and biological activity of boronate-modified ASOs and controls. (A) Data from chloroalkane penetration assay using HaloTag-expressing HeLa cells.¹⁶ ct-ASOs and control small molecule ct-W were incubated with cells at indicated concentrations for 4 h, followed by washes and a chase with ct-dye. CP₅₀ values were calculated based on the curve fits shown.¹⁷ Error bars and CP₅₀ errors are standard errors of the mean from at least three independent trials. (B) Schematic showing splice-correction based luciferase reporter assay.¹⁹ (C) Data from splice-correcting reporter assay using HeLa cells.¹⁷ All data are reported in terms of fold change of relative luminescence units (RLU) compared to untreated cells. Error bars show standard errors of the mean from three independent trials.