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.
Copyright Owner
Springer Science+Business Media, LLC, part of Springer Nature
Copyright Date
2018
Language
en
File Format
application/pdf
Recommended Citation
Singh, Natalia N.; Luo, Diou; and Singh, Ravindra N., "Pre-mRNA Splicing Modulation by Antisense Oligonucleotides" (2018). Biomedical Sciences Publications. 64.
https://lib.dr.iastate.edu/bms_pubs/64
Included in
Congenital, Hereditary, and Neonatal Diseases and Abnormalities Commons, Genetic Processes Commons, Genetics Commons
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.