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Article

Therapeutic Potential of Splice-Switching Oligonucleotides

6 Jan 2009

Abstract excerpt

Alternative splicing enables a single pre-messenger RNA transcript to yield multiple protein isoforms, making it a major contributor to the diversity of the proteome. While this process is essential for normal development, aberrations in alternative splicing are the cause of a multitude of human diseases. Methods for manipulating alternative splicing would thus be of therapeutic value. Chemically modified...

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Identifiers

PMID: 19125639
DOI: 10.1089/oli.2008.0161

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