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

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Restoration of Dystrophin Protein Expression by Exon Skipping Utilizing CRISPR-Cas9 in Myoblasts Derfor 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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In Vivo Evaluation of Dystrophin Exon Skipping in , Miceinvolves 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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Exon 51 Skipping Quantification by Digital Droplet PCR in del52hDMD/, Mice 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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https://doi.org/10.1007/978-3-031-14406-6ations, and its evolution into the approach we are now familiar with. We give a more extensive history of exon skipping in particular, as it is the splice modulation approach given the most focus in this book.

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Giordano Zambelli,Luciano Morganting authorization by the US Food and Drug Administration (FDA), on the condition that additional postapproval trials show clinical benefit. Permanent exon skipping achieved at the DNA level using clustered regularly interspaced short palindromic repeats (CRISPR) technology holds promise in current pr

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