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. 2019 May 25;47(14):7618–7632. doi: 10.1093/nar/gkz469

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© The Author(s) 2019. Published by Oxford University Press on behalf of Nucleic Acids Research.

This is an Open Access article distributed under the terms of the Creative Commons Attribution License (http://creativecommons.org/licenses/by/4.0/), which permits unrestricted reuse, distribution, and reproduction in any medium, provided the original work is properly cited.

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Figure 2. — Tissue distribution of AAV9 DNA and ExspeU1sma RNA expression. (A) SMA mild mice (smn^−/−; SMN2^2TG/2TG) were treated with AAV9-ExspeU1sma (n = 3), AAV9-U1wt (n = 3) and saline (n = 2) with two intraperitoneal injections (P0 and P2) at 1.5 × 10¹² vgp/mouse/injection. Animals were sacrificed and organs collected at P7. Data are expressed as mean ± SD of VCN per cell. Statistical analysis was performed using Two-Way ANOVA. Level of ExspeU1sma expression in (B) SMA mild (smn^−/−; SMN2^2TG/2TG) and (C) SMA severe (smn^−/−; SMN2^2TG/0) mice treated with saline and with AAV9-ExSpeU1sma (two intraperitoneal injections at P0 and P2, 1.5 × 10¹² vgp/mouse/injection). Data (mean ± SD) are expressed as percentage of ExSpeU1sma relative to endogenous U1. For each tissue statistical analysis, performed using Student’s t-test compares saline to U1sma-treated mice at P7 (n = 3 per genotype) (***P < 0.001; **P < 0.01; *P < 0.05; ns, not significant).