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. 2019 Mar 7;9:3807. doi: 10.1038/s41598-019-40421-z

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© The Author(s) 2019

Open Access This article is licensed under a Creative Commons Attribution 4.0 International License, which permits use, sharing, adaptation, distribution and reproduction in any medium or format, as long as you give appropriate credit to the original author(s) and the source, provide a link to the Creative Commons license, and indicate if changes were made. The images or other third party material in this article are included in the article’s Creative Commons license, unless indicated otherwise in a credit line to the material. If material is not included in the article’s Creative Commons license and your intended use is not permitted by statutory regulation or exceeds the permitted use, you will need to obtain permission directly from the copyright holder. To view a copy of this license, visit http://creativecommons.org/licenses/by/4.0/.

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Successful evaluation of exon-skipping in urine-derived cells (UDCs) from patients with exon 45–54 deletions. RT-PCR analysis of DYSTROPHIN after phosphorodiamidate morpholino oligomer (PMO) treatment in (A) 3-deazaneplanocin A hydrochloride (DZNep)-treated MYOD1-UDCs and (B) MYOD1-converted fibroblasts (MYOD1- Fibs) derived from a Duchenne muscular dystrophy (DMD) patient with an exon 45–54 deletion. DZNep-treated MYOD1-UDCs and MYOD1- Fibs were also treated with control antisense at 5 μM as controls. The upper bands are unskipped products (Δ45–54) that remain out of the reading frame. The lower bands are exon 44-skipped products (Δ44–54) that restore the open reading frame. Skipping efficiency was calculated as (exon 44-skipped transcript molarity)/(native + exon 44-skipped transcript molarity (marked with arrows)) ×100% using MultiNA. One-way ANOVA followed by Bonferroni’s post hoc test was used to compare the skipping efficiencies; n = 3, for each group; ****P < 0.0001. Data are expressed as mean ± SEM. PMO 44: PMO for skipping exon 44. (C,D) Immunoblotting for DYSTROPHIN in DZNep-treated MYOD1-UDCs and MYOD1-Fibs from the DMD patient after PMO 44 treatment. For DYSTROPHIN detection, anti-dystrophin AB15277 (against C-terminal) was used. The relative intensities of the bands normalized to α-Tubulin expression were compared in patient-derived cells with and without PMO treatment by performing a one-way ANOVA followed by Bonferroni’s post hoc test; n = 3, for each. **P < 0.01, ***P < 0.001. HI: healthy individual. (E,F) Immunocytochemistry for DYSTROPHIN in the DZNep-treated MYOD1-UDCs and MYOD1-Fibs after PMO 44 treatment. Representative pictures are shown. Scale bar: 100 μm.