Antisense‐Oligonucleotide – ein universelles Therapieprinzip

Abstract

AbstractIn recent years, a nova1 class of pharmaceuticals has been developed which bind in a predictable way to certain nucleic acid target sequences aiming at selective inhibition of expression of disease‐causing genes. 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 antisense oligonucleotide function, the structure of antisense oligonucleotide analogues, the different strategies for improvement of their biological potency, and selected reports on successful in vivo studies and first clinical investigations using these antisense oligonucleotides are discussed.