CURRENT POTENTIAL OF ANTISENSE OLIGONUCLEOTIDES AS THERAPEUTIC DRUGS

Antisense oligonucleotides have great potential as rationally designed therapeutic drugs by taking advantage of the basic Watson-Crick base pairing of nucleic acids. Such oligonucleotides may block synthesis of a specific protein, such a c-myb, and prevent smooth muscle prolifera tion involved in restenosis. Although promising, the technology has so me major hurdles to overcome before fruition. These include obtaining a stable backbone that can enter cells readily, overcoming problems of chirality, and solving the problems of delivery and metabolism. Altho ugh there are many reports of successful experiments using antisense o ligonucleotides, one must always keep in mind the complex nature of th ese experiments, as well as nonspecific effects that may masquerade as antisense effects.