Pharmacology of Antisense Therapeutic Agents: Cancer and Inflamination

Antisense oligonucleotides represent a new paradigm for drug discovery that holds great promise to deliver potent and specific drugs with fewer undesired side effects. The antisense paradigm offers the opportunity to identify rapidly lead compounds based on knowledge of the biology of a disease process, and a relevant target gene sequence. With this information, the practitioner of antisense drug discovery can rapidly design, synthesize, and test a series of compounds in cell culture and determine if the target gene is specifically inhibited. A compound thus identified can then be tested in an animal model, either to determine whether targeted gene expression can be inhibited in various animal tissues or to determine if there is activity in an animal model of a human disease. The length of time and the resources required to identify a lead compound by the antisense paradigm is much less than by any other drug discovery method.