Journal or Book Title
Exon Skipping and Inclusion Therapies
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.
Springer Science+Business Media, LLC, part of Springer Nature
Singh, Natalia N.; Luo, Diou; and Singh, Ravindra N., "Pre-mRNA Splicing Modulation by Antisense Oligonucleotides" (2018). Biomedical Sciences Publications. 64.