THERAPEUTIC POTENTIAL AND MECHANISM OF ACTION OF OLIGONUCLEOTIDES ANDRIBOZYMES

Specific inactivation of gene expression is an attractive approach for rational drug design to combat degenerative diseases and infectious a gents. Oligonucleotide-directed triple-helix formation at cis-acting e lements of gene promoters, short oligonucleotides containing base sequ ences that are complementary to the messenger RNA (antisense oligos), and RNA enzymes (ribozymes) that specifically cleave messenger RNA mol ecules are currently being used both as experimental tools and as ther apeutic agents. Mechanisms of action of various oligonucleotide-based drugs, recent developments in the drug-delivery approaches, and future potentials are discussed in this review. (C) 1997 Academic Press.