Titlebook: Exon Skipping and Inclusion Therapies; Methods and Protocol Toshifumi Yokota,Rika Maruyama Book 2018 Springer Science+Business Media, LLC,

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J. Nagarjun,S. Manimaran,M. KrishnaprakashDuchenne muscular dystrophy (DMD). Systemic administration of antisense phosphorodiamidate morpholino oligomers (PMOs) targeting exons 6 and 8 in dystrophin mRNA of the canine X-linked muscular dystrophy model in Japan (CXMD.) that lacks exon 7, restored dystrophin expression throughout skeletal mus

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https://doi.org/10.1007/978-3-540-88087-5for myofiber integrity. Exon skipping therapy is an emerging strategy for restoring the open reading frame of the . gene to produce functional protein in DMD patients by skipping single or multiple exons. Although antisense oligonucleotides are able to target pre-mRNA for exon skipping, their half-l

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Fuzziness in Information Systemsinvolves the following two aspects: (1) efficiency and accuracy of exon skipping and levels of dystrophin expression determined by RT-PCR, immunochemistry, and western blotting; (2) therapeutic effects on muscle pathology and functions assessed by histology and functional assays including grip stren

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Studies in Systems, Decision and Control protein. Antisense oligonucleotide (AON)-mediated exon skipping has been developed as a method to restore the reading frame, which allows the synthesis of internally truncated, but partially functional dystrophin proteins, as found in the less severe Becker muscular dystrophy (BMD). This approach i

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Toshifumi Yokota,Rika MaruyamaIncludes cutting-edge methods and protocols.Provides step-by-step detail essential for reproducible results.Contains key notes and implementation advice from the experts

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