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Methods Mol Biol. 2014;1126:271-83. doi: 10.1007/978-1-62703-980-2_20.

Antisense methods to modulate pre-mRNA splicing.

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Department of Biomedical Sciences, College of Veterinary Medicine, Iowa State University, Ames, IA, USA.


The dynamic process of pre-mRNA splicing is regulated by combinatorial control exerted by overlapping cis-elements that are unique to every exon and its flanking intronic sequences. Splicing cis-elements are usually 4-8-nucleotide-long linear motifs that furnish interaction sites for specific proteins. Secondary and higher-order RNA structures exert an additional layer of control by providing accessibility to cis-elements. Antisense oligonucleotides (ASOs) that block splicing cis-elements and/or affect RNA structure have been shown to modulate alternative splicing in vivo. Consistently, ASO-based strategies have emerged as a powerful tool for therapeutic manipulation of aberrant splicing in pathological conditions. Here we describe the application of an ASO-based approach for the enhanced production of the full-length mRNA of SMN2 in spinal muscular atrophy patient cells.

[Indexed for MEDLINE]

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