ABSTRACT
This chapter assesses the status of the technology, discusses the technology in the pharmacological context in which it is best understood, and deals with some of the controversies with regard to the technology and the interpretation of experiments. Interest in developing antisense technology and in exploiting it for therapeutic purposes has been intense. Phosphorothioate oligonucleotides can be prepared consistently and with excellent purity. The structure of the Ribonucleic acid (RNA) has a profound influence on the affinity of the oligonucleotide and on the rate of binding of the oligonucleotide to its RNA target. Clearly, the ultimate biological effect of an oligonucleotide will be influenced by the local concentration of the oligonucleotide at the target RNA, the concentration of the RNA, the rates of synthesis and degradation of the RNA, the type of terminating mechanism, and the rates of the events that result in termination of the RNA’s activity.
