ANTISENSE OLIGONUCLEOTIDES AS THERAPEUTIC AGENTS

The potential for modulating gene expression by the use of antisense o ligonucleotides has become increasingly interesting in recent years. A ntisense oligonucleotides are complementary nucleic acid fragments tha t hybridize to target sequences within RNA to form a DNA-RNA duplex, r esulting in the block of translation of messenger RNA into the protein . Advances in chemistry and molecular biology have provided the basis to develop antisense oligodeoxynucleotides and improve their selectivi ty, stability and specificity of action. The antisense technology has been extensively used in vitro and in vivo as a tool to study the regu latory mechanisms in biologic processes and as potential therapeutic a gents in cancer, viral infections and genetic disorders. In the presen t review, the various approaches for the use of antisense molecules in oncology, virology, genetic and inflammatory diseases are described; several studies, supporting the in vitro and in vivo applications of t his technology, are also presented. Moreover, the potential clinical u se of antisense therapies is discussed. (C) 1997 The Italian Pharmacol ogical Society.