Pre-mRNA Splicing Modulation by Antisense Oligonucleotides

Methods in Molecular Biology
Natalia N SinghRavindra N Singh

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.

Citations

May 7, 2020·Frontiers in Chemistry·Nihay Laham-KaramPiia Kokkonen
Aug 7, 2019·Cellular and Molecular Neurobiology·Benedetta PerroneSebastiano Cavallaro
Dec 8, 2020·Neuroscience Insights·Ravindra N SinghNatalia N Singh
Dec 15, 2020·Trends in Biochemical Sciences·Thi Khanh LePalma Rocchi
Jun 20, 2021·Nature Reviews. Drug Discovery·Melanie WinkleGeorge A Calin
Aug 28, 2021·International Journal of Molecular Sciences·Eric William OttesenRavindra Narayan Singh

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