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Curr Opin Mol Ther. 2005 Oct;7(5):476-82.

Making antisense of splicing.

Author information

1
Duke University Medical Center, Department of Molecular Genetics and Microbiology, Center for RNA Biology, Durham, NC 27710, USA. garci001@mc.duke.edu

Abstract

Alternative splicing multiplies the coding capacity of the genome, resulting in an expanded proteome that provides many targets for therapy. In addition to creating this diverse pharmacoproteome, the process of splicing can be targeted by conventional and molecular therapies. Splicing as a therapeutic target is highlighted in this review, with a particular emphasis on oligonucleotide-based molecular approaches. These oligonucleotides can be used to promote skipping of constitutive exons, inhibit inappropriately activated exons, or stimulate exons weakened by mutations. Preliminary, but exciting, results suggest that these reagents could have clinical utility in treating previously intractable conditions.

PMID:
16248283
[Indexed for MEDLINE]

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