Chapter title |
Pre-mRNA Splicing Modulation by Antisense Oligonucleotides
|
---|---|
Chapter number | 26 |
Book title |
Exon Skipping and Inclusion Therapies
|
Published in |
Methods in molecular biology, September 2018
|
DOI | 10.1007/978-1-4939-8651-4_26 |
Pubmed ID | |
Book ISBNs |
978-1-4939-8650-7, 978-1-4939-8651-4
|
Authors |
Natalia N. Singh, Diou Luo, Ravindra N. Singh, Singh, Natalia N., Luo, Diou, Singh, Ravindra N. |
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. |
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