Link between MHC Fiber Type and Restoration of Dystrophin Expression and Key Components of the DAPC by Tricyclo-DNA-Mediated Exon Skipping
نویسندگان
چکیده
منابع مشابه
Relationship between myosin heavy chain fibre type and restoration of dystrophin expression and key components of the dystrophin-associated glycoprotein complex by Tricyclo-DNA mediated exon skipping
Exon skipping mediated by tricyclo-DNA (tc-DNA) antisense oligonucleotides has been shown to induce significant levels of dystrophin restoration in mdx, a mouse model of Duchenne Muscular Dystrophy. This translates into significant improvement in key disease indicators in muscle, cardio-respiratory function, heart and the central nervous system. Here we examine the relationship between muscle f...
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Restoration of dystrophin expression in mdx muscle cells by chimeraplast-mediated exon skipping.
The most common types of dystrophin gene mutations that cause Duchenne muscular dystrophy (DMD) are large deletions that result in a shift of the translational reading frame. Such mutations generally lead to a complete absence of dystrophin protein in the muscle cells of affected individuals. Any therapeutic modality that could restore the reading frame would have the potential to substantially...
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ژورنال
عنوان ژورنال: Molecular Therapy - Nucleic Acids
سال: 2017
ISSN: 2162-2531
DOI: 10.1016/j.omtn.2017.10.014