Publication: Therapeutic potential of splice-switching oligonucleotides
Issued Date
2009-03-01
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ISSN
15454576
Other identifier(s)
2-s2.0-61349157969
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Mahidol University
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SCOPUS
Bibliographic Citation
Oligonucleotides. Vol.19, No.1 (2009), 1-13
Suggested Citation
John Bauman, Natee Jearawiriyapaisarn, Ryszard Kole Therapeutic potential of splice-switching oligonucleotides. Oligonucleotides. Vol.19, No.1 (2009), 1-13. doi:10.1089/oli.2008.0161 Retrieved from: https://repository.li.mahidol.ac.th/handle/20.500.14594/27275
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Title
Therapeutic potential of splice-switching oligonucleotides
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Abstract
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 antisense oligonucleotides that alter alternative splicing by directing splice site selection have been developed to achieve this end. These splice-switching oligonucleotides (SSOs) have been applied to correct aberrant splicing, induce expression of a therapeutic splice variant, or induce expression of a novel therapeutic splice variant in a number of disease-relevant genes. Recently, in vivo efficacy of SSOs has been reported using animal disease models, as well as in results from the first clinical trial. © Mary Ann Liebert, Inc. 2009.