Antisense oligonucleotides: a novel therapeutic approach.

The use of antisense oligonucleotides as therapeutic agents has generated considerable enthusiasm in the research and medical community. Oligonucleotides inhibit gene expression by binding to their target nucleic acid with high specificity and selectivity. The field of antisense technology has progressed enormously. Major progress has been accomplished in the synthesis and manufacturing of modified oligonucleotides. Numerous studies have demonstrated the ability of antisense oligonucleotides to modulate gene expression, in such diverse fields as infectious diseases, cancer, and inflammation. More than a dozen of clinical trials using antisense oligonucleotides have been initiated during the last three years or so. The insights gained through these ongoing clinical trials has opened the pathway to the design of more advanced chemistries which have improved safety profile and efficacy.