siRNA as a new drug: intellectual property

Abstract

RNA interference (RNAi) is a powerful tool for gene function analysis and drug discovery research. In this process, small interfering RNA (siRNA) silences the expression of a specific protein, which is encoded by a nucleic acid sequence that is complementary to the sequence of the siRNA. The reproducibility, specificity and magnitude of the silencing effect have stimulated a fast adoption of this process as the method of choice to study loss-of-function phenotypes and validate drug targets. In addition, siRNA may offer promise as a new drug. For successful application as a therapeutic, stability in the biological environment and efficiency of target cell entry of siRNA need to be addressed. This review describes scientific literature and patents (applications) related to the use of RNAi and innovations on siRNA stability and invivo delivery to target cells.

• chemical modification
• double stranded RNA
• drug delivery
• gene silencing
• RNAinterference (RNAi)
• small interfering RNA (siRNA)