Antisense therapeutics (original) (raw)

Nature Biotechnology volume 17, pages 403–404 (1999)Cite this article

Antisense molecules are stretches of single-stranded nucleic acid that target and bind with a specific messenger RNA, interfering with and even preventing the translation or overexpression of the protein encoded by the mRNA. They are referred to as antisense because the "sense" sequence and orientation of an mRNA is the one that directly translates itself into protein, whereas "antisense" is the complementary strand that hybridizes with the mRNA and prevents it from carrying out its function. The ability to interfere with a critical point along the gene expression and protein synthesis pathway is what makes antisense such an attractive molecular therapeutic platform. In principle, antisense molecules can be applied to any disease in which protein overexpression or even gene activation is detrimental, and are now being used to dissect gene function as well as to develop therapeutics against a wide variety of diseases.

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  1. vice president for business development at Argonex Inc., 2044 India Road, Charlottesville, 22901, VA
    Aris Persidis

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Persidis, A. Antisense therapeutics.Nat Biotechnol 17, 403–404 (1999). https://doi.org/10.1038/7973

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