Delivery and targeting of oligonucleotides and siRNA

Oligodeoxynucleotides (ODNs) are increasingly being recognized as potential therapeutic agents to modulate aberrant gene expression for treating various diseases, including cancers1-3 and viral infections.4,5 Concerted efforts have made signicant progress in turning these nucleic acids into therapeutics. Apart from immune-stimulation and enzymatic cleavage, the most important feature of ODNs is their ability to block mRNA function by sequence-specic hybridization with target mRNA.6 Theoretically, the antisense strategy can be used to target any gene in the body, which allows these nucleic acids to achieve broader therapeutic potential than small molecules.