Extended Data Figure 5: ASO-5 reduces MECP2 mRNA and protein levels. | Nature

Extended Data Figure 5: ASO-5 reduces MECP2 mRNA and protein levels.

From: Reversal of phenotypes in MECP2 duplication mice using genetic rescue or antisense oligonucleotides

Extended Data Figure 5

a, Location of ASOs targeting sequences on the MECP2 pre-mRNA. Boxes represent exons, and lines denote introns. Boxes in blue denote the translatable regions. b, Section schemata from the Paxinos and Franklin35 mouse brain atlas showing site of stereotactic injection. c, Western blot (c) and densitometric analysis (d) of MeCP2 from cortical samples of mice treated with saline or the indicated ASOs 2 weeks after single bolus stereotactic injection of 500 μg ASO in the right ventricle of the brain. ASO-5 was found to be the most effective. GAPDH was used as an internal control (n = 3, one-way ANOVA followed by Tukey HSD post hoc correction for multiple comparisons; for gel source data, see Supplementary Fig. 4). e, RT–qPCR analysis of MECP2 mRNA from cortical samples of mice treated with saline or the indicated ASOs, 2 weeks after single bolus stereotaxic injection of 500 μg ASO in the right ventricle of the brain. ASO-5 was found to be the most effective. The MECP2 primers are common to the mouse and human alleles. Hprt1 was used as an internal control (n = 3, one-way ANOVA followed by Tukey HSD post hoc correction for multiple comparisons). Data are mean ± s.e.m. *P < 0.05; **P < 0.01; ***P < 0.001.

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