Challenges and Opportunities for Local and Systemic Delivery of siRNA and Antisense Oligonucleotides

The discovery and application of oligonucleotide technologies have already changed our fundamental understanding of biology and created a wealth of possibilities for development of new therapeutic entities. A major advantage of oligonucleotide-based technologies is the potential to develop specific therapeutic antagonists to approach targets previously considered undruggable with a traditional small-molecule approach. Although there is great potential for this technology in the development of new therapeutic agents, there are significant issues to be overcome before this technology can be readily translated into therapeutic use.