Are Antisense Oligonucleotides Therapeutic Agents of the Future

The idea of using sequence-specific oligonucleotides to inhibit the expression of individual genes and thereby cure a disease is a topic of great interest in the scientific as well as the financial communities. Most major pharmaceutical companies have a strong interest if not an active research group working on the topic. Numerous small “start-up” biotechnology companies have been organized in the past two or three years whose major or sole reason for existence is the development of oligonucleotides as therapeutic agents. Is this so-called “antisense” approach nonsense or is it realistic? Can we expect that anti-sense reagents will be the drugs of the future? An answer to these questions is not possible at this time. However, anti-sense is a fertile area of investigation. Much research and development is underway on many fronts. Clearly, insight into the feasability of anti-sense agents as therapeutic drugs will be forthcoming. In this article I will highlight some recent developments as well as bring out some of the problems and pitfalls encountered and the pursuits researchers are taking to circumvent these problems. Readers are referred to the recent reviews (1, 2, 3) and volumes (4, 5, 6) on antisense research for more specific details.