Running interference: an overview of therapeutic RNAi

RNA interference (RNAi) is a powerful, naturally occurring gene-silencing phenomenon. Our understanding of the mechanism has evolved rapidly, resulting in significant interest in its therapeutic potential. The power to specifically and efficiently silence any gene of interest hints at the power to approach clinically difficult problems in new and exciting ways. As examples, Huntington’s disease, prostate cancer, and HIV infection are examined briefly. Several key challenges currently face succesful translation of RNAi into viable therapeutics. These involve the human miRNA system, the chemistry of small interfering RNA (siRNA), and the development of suitable delivery strategies. Given the potential of the mechanism, and the impressive interest in RNAi for so many applications, it appears likely that RNAi will make a name for itself as a whole new therapeutic modality.