Oligonucleotide pharmacotherapy: an antigene strategy.

Oligonucleotide pharmacotherapy, although in a very preliminary stage, promises to provide new, highly specific tools for the treatment of human diseases, such as viral illnesses and cancer. The agents have several proposed mechanisms of action, including inhibition of translation, splicing, and transcription. In addition, the bioefficacy of oligonucleotides may be enhanced by phosphorothioates, methylphosphonates, and alpha-oligonucleotides. The agents are delivered by the ex vivo or topical route, and new methods of administration are under study. It is predicted that within the decade these agents will be used routinely to treat several serious illnesses.