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Making Sense of Antisense

As first complimentary oligonucleotide is poised for market, questions remain about mechanisms A drug that halts an AIDS-related eye infection could be the first antisense therapy to reach the market. However, whether the drug can truly be called "antisense" depends on some specifics--such as the ability to bind to targeted messenger RNA (mRNA) but avoid clinging to other proteins. Representatives of the drug's manufacturer, Isis Pharmaceuticals Inc. of Carlsbad, Calif., say fomivirsen (Vitra

Paul Smaglik


As first complimentary oligonucleotide is poised for market, questions remain about mechanisms
A drug that halts an AIDS-related eye infection could be the first antisense therapy to reach the market. However, whether the drug can truly be called "antisense" depends on some specifics--such as the ability to bind to targeted messenger RNA (mRNA) but avoid clinging to other proteins.

Representatives of the drug's manufacturer, Isis Pharmaceuticals Inc. of Carlsbad, Calif., say fomivirsen (Vitravene) excels at binding to mRNA. However, some antisense researchers outside the company caution that, in the past, antisense molecules thought to be highly specific have latched onto other proteins. A U.S. Food and Drug Administration advisory committee, which recently recommended that the drug be approved for marketing, only weighs in on the safety and efficacy of the drug, not the mechanism by which it works.

If that mechanism is, indeed, truly antisense--that is, works primarily through a...

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