ABSTRACT
Oligonucleotides represent a novel class of therapeutics with unique mechanisms of action. A typical oligonucleotide is a short chain DNA or RNA molecule, usually manufactured by chemical synthesis utilizing automated synthesizers. Although most oligonucleotides in clinical development are chemically similar, DNA, RNA, or LNA, often with chemical modi‰cations to afford nuclease stability in biological matrices, the mechanisms of action vary widely. As a consequence, a broad range of oligonucleotides are currently in clinical development for a variety of clinical indications. The types of oligonucleotides in preclinical or clinical development include antisense oligonucleotides, immunostimulatory oligonucleotides, DNA duplex decoys, small interfering RNAs (siRNAs), ribozymes, microRNAs, aptamers, and spiegelmers.
