Human therapeutics based on triple helix technology

Abstract

The application of triple helix technology to rational drug discovery is rapidly leading to the development of a new class of drugs, initially applicable for the effective and specific treatment of viral infections and, eventually, perhaps, cancer and immunological disorders. Issues such as stability, delivery, specificity, in vitro efficacy and acute toxicity, and the cost of synthesis are already being addressed: preliminary studies are yielding promising results. Further chemical modification of the oligonucleotides will probably be necessary to enhance affinity and efficacy, and comprehensive studies on the pharmacokinetics, toxicity, mutagenicity and in vivo efficacy of these compounds are still required. These, as well as scale-up synthesis and pharmaceutical formulation issues, are the focus of the R&D programs of a number of pharmaceutical laboratories.