Pharmacokinetic Properties of Phosphorothioate Oligonucleotides in Humans

The use of antisense molecules to inhibit the expression of disease-causing proteins represents a new paradigm in disease treatment. The molecular target for the antisense molecules, mRNA, is chemically and biologically well defined. This well-defined target is the same for all different possible therapeutic applications, whether the application is antiviral, anticancer, or anti-inflammatory. The specificity with which antisense molecules are capable of inhibiting gene expression, exemplified by the isozyme selectivity demonstrated by [Dean and Mckay 1994], has allowed entirely novel molecular targets to be explored for potential therapeutic applications. Thus antisense molecules have presented an opportunity to exploit a single target for multiple therapeutic indications as well as to explore the utility of inhibiting targets which traditional therapeutic agents could not specifically inhibit.