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

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An Overview of Recent Advances and Clinical Applications of Exon Skipping and Splice Modulation for proach 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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Recent Advances and Clinical Applications of Exon Inclusion for Spinal Muscular Atrophytein. 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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Nusinersen in the Treatment of Spinal Muscular Atrophyss of motor neurons. With the discovery of the intronic splicing silencer N1 (ISS-N1) as a potential target for antisense therapy, several antisense oligonucleotides (ASOs) are being developed to include exon 7 in the final mRNA transcript of the . gene and thereby increasing the production of spina

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