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