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. 2013 Jan 15;21(3):602–609. doi: 10.1038/mt.2012.264

Figure 4.

Figure 4

Exon skipping in transgenic human APOB mice. Mice were injected intravenously via the tail vein with IVF2.0-formulated SSO at the indicated doses and killed at the indicated times for collection of tissue samples from liver. RNA was extracted and skipping efficiencies were determined by RT-qPCR. (a) Dose escalation study with SSO 14-53PF. Samples taken 24 hours after injection: 12.5 mg/kg (5.80 ± 3.65%), 25 mg/kg (29.07 ± 8.30%), 50 mg/kg (20.40 ± 11.80%) and vehicle only control consisting of IVF2.0 without complexed SSO (0.39 ± 0.13%). (b) Time course after single injection with 25 mg/kg of SSO 14-53PF: 24 hour (29.07 ± 8.30%, n = 5), 48 hour (22.04%, n = 2), 72 hour (20.57%, n = 2), and 6 days (7.55 ± 3.13%, n = 5). (c) SSO comparison, samples taken at 6 days after injection, n = 3 each group: 14-53P (5.95 ± 1.13%), 14-53PF (5.39 ± 0.35%), sc14-53P (scrambled control; 0.5 ± 0.05%), 1-F (0.72 ± 0.21%), and 14-F (0.72 ± 0.26%). (d) Quantification of APOB26–27 (27 inc), APOB26–28 (skip), APOB25–26 (total), and mouse Apob (ms) in mice with successful treatment (SSO 14-53P and 14-53PF) relative to control/unsuccessful treatment (SSO sc14-53PF/1-F and 14-F), presented as whisker plots, where the line within the box marks the median value, the box denotes the interquartile range (25th–75th centile) and the whiskers show the maximum and minimum observations. IVF2.0, Invivofectamine 2.0; RT-qPCR, reverse transcriptase-quantitative polymerase chain reaction; SSO, splice-switching oligonucleotides.