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Abstract
An estimated 60% of all human genes undergo alternative splicing, a highly regulated process that produces splice variants with different functions. Such variants have been linked to a variety of cancers, and genetic diseases such as thalassemia and cystic fibrosis. This Perspective describes a promising approach to RNA repair based on the use of antisense oligonucleotides to modulate alternative splicing and engender the production of therapeutic gene products.
Authors
Peter Sazani, Ryszard Kole
Figure 1
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Modification of splicing by antisense oligonucleotides. Aberrant splicing in thalassemic β-globin pre-mRNA or in certain splice mutants in CFTR is prevented, and correct splicing is restored, by oligonucleotides (dark red bars) that block aberrant 5′ or 3′ cryptic splice sites (a ). Similarly, oligonucleotides induce skipping of a normal exon (gray) (b ) or force selection of an alternative 5′ splice site (c ) by antisense oligonucleotides targeted to appropriate splice sites.
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ISSN: 0021-9738 (print), 1558-8238 (online)