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Antisense therapy is a theoretical form of treatment for genetic disorders or infections. When the genetic sequence of a particular gene is known to be causative of a particular disease, it is possible to synthesize a strand of nucleic acid (DNA, RNA or a chemical analogue) that will bind to the messenger RNA produced by that gene, effectively turning that gene "off".

This synthesized nucleic acid is termed an "anti-sense" oligo because its base sequence is complementary to the gene's messenger RNA (mRNA), which is called the "sense" sequence (so that a sense segment of mRNA " 5'-AAGGUC-3' " would be blocked by the anti-sense mRNA segment " 3'-UUCCAG-5' ").

Most potential therapies have not yet produced significant clinical results, though one antisense drug, Formivirsen (marketed as Vitravene), has been approved by the US Food and Drug Administration (FDA).

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Applied genetics | Biotechnology

 

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