THE ANTISENSE OLIGONUCLEOTIDE - A UNIVERSAL THERAPEUTIC PRINCIPLE

In recent years, a noval class of pharmaceuticals has been developed w hich bind in a predictable way to certain nucleic acid target sequence s aiming at selective inhibition of expression of disease-causing gene s. The chemical structure of these so-called antisense oligonucleotide compounds must be altered relative to their natural models to render them stable under in vivo conditions and to allow their penetration to the site of action inside cells. In this article, the principle of an tisense oligonucleotide function, the structure of antisense oligonucl eotide analogues, the different strategies for improvement of their bi ological potency, and selected reports on successful in vivo studies a nd first clinical investigations using these antisense oligonucleotide s are discussed.