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CropBox PageLayout: SinglePage PageMode: UseNone Language: en-US Outlines: Item: Title: AntimiR treatment corrects myotonic dystrophy primary cell defects across several CTG repeat expansions with a dual mechanism of action Children: Item: Title: INTRODUCTION Item: Title: RESULTS Children: Item: Title: Characterization of myoblast cell lines from patients with adult- and juvenile-onset DM1 Item: Title: Reduction of miR-23b and miR-218 overexpression during DM1 myotube differentiation by antimiR treatment Item: Title: Reduction of MBNL1 levels during differentiation of DM1 myotubes and their increase upon antimiR treatment Item: Title: Reduction in the number of RNA and MBNL1 nuclear foci and DMPK transcripts upon antimiR treatment Item: Title: Correction of aberrant splicing in DM1 myoblasts Item: Title: Functional rescue of myotubes area and fusion index Item: Title: Integrated evaluation of the antimiR’s therapeutic potential Item: Title: RNA-seq analysis of primary cells treated with antimiR-23b-V2 Item: Title: DISCUSSION Item: Title: MATERIALS AND METHODS Children: Item: Title: Study design Item: Title: Sample donor characterization Item: Title: Primary myoblast isolation from muscle biopsy Item: Title: CTG expansion sizing Item: Title: DMPK expression quantification by RT-qPCR Item: Title: Protein extraction and Jess Simple Western Item: Title: Statistical analyses Item: Title: Supplementary Materials Children: Item: Title: This PDF file includes: Item: Title: REFERENCES AND NOTES Item: Title: Acknowledgments Info: Title: Science Journals   AAAS Creator: Adobe InDesign 19.5 (Windows) Producer: Adobe PDF Library 17.0 CreationDate: Tue Sep 24 02:50:06 CEST 2024 ModDate: Tue Sep 24 02:50:08 CEST 2024 ID: 0x088712275ff06f408805bea313f5b8b6, 0xc3388a78af3b3741b8da0e5609717eee Filters: FilterPipeline: FlateDecode Images: Image: NisoImageMetadata: FormatName: image/png ImageWidth: 1506 ImageHeight: 3355 BitsPerSample: 8 BitsPerSampleUnit: integer Filter: 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