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

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https://doi.org/10.1007/978-3-031-14406-6ase. Defects in RNA splicing account for at least 10% of all genetic disorders, with the number expected to increase as more information is uncovered on the contribution of noncoding genomic regions to disease. Splice modulation through the use of antisense oligonucleotides (AOs) has emerged as a pr

MUT

Giordano Zambelli,Luciano Morgantiproach uses antisense oligonucleotides (AON) to modify the splicing of pre-mRNA to correct the mutation responsible for a disease, or to suppress a particular gene expression, as in allergic diseases. Antisense-mediated exon skipping is most extensively studied in Duchenne muscular dystrophy (DMD) a

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Ultrasound, Embodiment and Abortion,tein. Insufficient levels of SMN results in the loss of motor neurons, which causes muscle weakness, respiratory distress, and paralysis. A nearly identical gene (.) contains a C-to-T transition which excludes exon 7 from 90% of the mature mRNA transcripts, leading to unstable proteins which are tar

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Critical