ABSTRACT
The use of antisense oligonucleotides as therapeutic agents represents a new paradigm in drug discovery and development (1-6). Antisense drugs, which are short DNA oligonucleotide strands, are designed to inhibit the production of disease-causing proteins and can be used to treat a wide range of infectious, inflammatory, and cardiovascular diseases as well as cancer. The antisense mechanism of action is shown schematically in Figure 1. Because of their high degree of selectivity for the intended biological target, oligonucleotide drugs have the potential to be effective and less toxic than traditional drugs. For these reasons, the past decade has seen explosive growth in the areas of oligonucleotide chemistry and biology toward identification of leading drug candidates (7-10).
