Therapeutic antisense and ribozymes

Abstract

The regulation of expression of genetic information by complementary pairing of sense and antisense nucleic acid strands has been termed 'antisense', and is a mechanism used throughout nature to regulate gene expression. It is now possible to design antisense DNA oligonucleotides, or catalytic antisense RNAs (ribozymes) which can pair with and functionally inhibit the expression of any single stranded nucleic acid in a sequence specific fashion. This high degree of specificity has made them attractive candidates for therapeutic agents. These molecules have the potential for the treatment of a wide variety of diseases. The recent development of retroviral as well as other viral and non-viral based delivery schemes make the clinical use of these molecules a virtual certainty.