Chapter 8 Antisense RNA and DNA as potential therapeutic agents

Abstract

Summary It is evident that antisense technology has practical potential in terms of biotechnological and pharmacological applications. Several biotechnology companies have antisense strategies as a primary focus, and several established pharmaceutical companies have initiated antisense research and development programs. Antisense technology is already proving extremely useful to the biotechnology industry intersted in commercial plant development; e.g., tomatoes that ripen more slowly and tobacco plants that are more resistant to virus infections. However, it is becoming increasingly clear that antisense strategies are not as straightforward or as easily applicable to the whole animal or human as had been suggested in early experiments with cultured cells. Part of the problem is that the exact mechanisms of gene inhibition are not known in molecular terms for each class of antisense agents. Therefore, the specificity and extent of gene inhibition are difficult to predict. A number of other problems related to specificity, drug delivery, and pharmacokinetics will also need to be overcome to allow the rational design of antisense therapeutic strategies. Nevertheless, the rapidly expanding database of knowledge concerning the molecular mechanism of action of antisense inhibition of gene expression, and the continual development of new classes of selectively modified antisense compounds hold great promise that antisense therapeutics for human disease will be an important component of 21st century medicine.