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Review
. 2009 Apr;11(2):116-23.

Progress toward therapy with antisense-mediated splicing modulation

Liutao Du  1 , Richard A Gatti
Affiliations
Review

Progress toward therapy with antisense-mediated splicing modulation

Liutao Du et al. Curr Opin Mol Ther. 2009 Apr.

Abstract

Antisense oligonucleotides (AO) or antisense RNA can complementarily bind to a target site in pre-mRNA and regulate gene splicing, either to restore gene function by reprogramming gene splicing or to inhibit gene expression by disrupting splicing. These two applications represent novel therapeutic strategies for several types of diseases such as genetic disorders, cancers and infectious diseases. In this review, the recent developments and applications of antisense-mediated splicing modulation in molecular therapy are discussed, with emphasis on advances in antisense-mediated splice targeting, applications in diseases and systematic delivery.

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Figures

Figure 1
Figure 1. Schematic demonstration of antisense-mediated splicing modulation
(A) Antisense oligonucleotide (AO) blocking of a mutation-induced cryptic 5′ splicing site (SS). (B) AO blocking of exon-intron junctions and/or exonic splicing enhancer (ESE) site to induce exon skipping. (C) AO blocking of exonic splicing silencer (ESS) or intronic splicing silencer (ISS) sites to enhance exon inclusion.
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