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Curr Opin Mol Ther. 2009 Apr;11(2):116-23.

Progress toward therapy with antisense-mediated splicing modulation.

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Department of Pathology and Laboratory Medicine, The David Geffen School of Medicine at UCLA, 675 Charles Young Drive South, CA 90095-1732, USA.


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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