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 proliferation involved in restenosis. Although promising, the technology has some 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. Although there are many reports of successful experiments using antisense oligonucleotides, one must always keep in mind the complex nature of these experiments, as well as nonspecific effects that may masquerade as antisense effects.