RNA directed therapeutics: mechanisms and status

Progress in antisense technology has identified multiple post hybridization mechanisms that have been successfully exploited to produce pharmacological effects in cells, animals and humans. Advances in understanding the pharmacokinetics and toxicological properties of various types of oligoneucleotides have also been reported. Together these data support optimism that RNA-based therapeutics may bring value. In this very brief review, I focus on the advances in understanding the molecular mechanisms by which antisense drugs work with particular focus on RNase H1 and RISC mediated target reduction.