The use of antisense oligonucleotides as therapeutic agents has genera
ted considerable enthusiasm in the research and medical community. Oli
gonucleotides inhibit gene expression by binding to their target nucle
ic acid with high specificity and selectivity. The field of antisense
technology has progressed enormously. Major progress has been accompli
shed in the synthesis and manufacturing of modified oligonucleotides.
Numerous studies have demonstrated the ability of antisense oligonucle
otides to modulate gene expression, in such diverse fields as infectio
us diseases, cancer, and inflammation. More than a dozen of clinical t
rials using antisense oligonucleotides have been initiated during the
last three years or so. The insights gained through these ongoing clin
ical trials has opened the pathway to the design of more advanced chem
istries which have improved safety profile and efficacy.