Antisense Oligonucleotides as Therapeutics and their Delivery

Abstract

Antisense oligonucleotides are novel, highly selective inhibitors or modulators of gene expression. Attention is now being paid for the use of these agents not only in the treatment of genetic disorders but also for nongenetic disorders, where the treatment involves modulation of gene expression. However, antisense oligonucleotides face immense challenges for their use as therapeutics and successful application of these agents requires appropriate design and delivery strategies to increase their stability and intracellular uptake.