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Contribution to Book
Pre-mRNA Splicing Modulation by Antisense Oligonucleotides
Exon Skipping and Inclusion Therapies
  • Natalia N. Singh, Iowa State University
  • Diou Luo, Iowa State University
  • Ravindra N. Singh, Iowa State University
Document Type
Article
Publication Version
Accepted Manuscript
Publication Date
1-1-2018
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
Citation Information
Natalia N. Singh, Diou Luo and Ravindra N. Singh. "Pre-mRNA Splicing Modulation by Antisense Oligonucleotides" Exon Skipping and Inclusion Therapies Vol. 1828 (2018) p. 415 - 437
Available at: http://works.bepress.com/ravindra-singh/37/