Campus Units

Biomedical Sciences

Document Type

Article

Publication Version

Accepted Manuscript

Publication Date

2018

Journal or Book Title

Exon Skipping and Inclusion Therapies

Volume

1828

First Page

415

Last Page

437

DOI

10.1007/978-1-4939-8651-4_26

Abstract

Pre-mRNA splicing, a dynamic process of intron removal and exon joining, is governed by a combinatorial control exerted by overlapping cis-elements that are unique to each exon and its flanking intronic sequences. Splicing cis-elements are usually 4-to-8-nucleotide-long linear motifs that provide binding sites for specific proteins. Pre-mRNA splicing is also influenced by secondary and higher order RNA structures that affect accessibility of splicing cis-elements. Antisense oligonucleotides (ASOs) that block splicing cis-elements and/or affect RNA structure have been shown to modulate splicing in vivo. Therefore, ASO-based strategies have emerged as a powerful tool for therapeutic manipulation of splicing in pathological conditions. Here we describe an ASO-based approach to increase the production of the full-length SMN2 mRNA in spinal muscular atrophy patient cells.

Comments

This is a post-peer-review, pre-copyedit version of an article published as Singh N.N., Luo D., Singh R.N. (2018) "Pre-mRNA Splicing Modulation by Antisense Oligonucleotides." In: Yokota T., Maruyama R. (eds) Exon Skipping and Inclusion Therapies. Methods in Molecular Biology, vol 1828. Humana Press, New York, NY. The final authenticated version is available online at DOI: 10.1007/978-1-4939-8651-4_26. Posted with permission.

Copyright Owner

Springer Science+Business Media, LLC, part of Springer Nature

Language

en

File Format

application/pdf

Published Version

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