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Mission RNAi Management Team Board of Directors Scientific Advisory Board

RNA Interference (RNAi)

Ribonucleic acid (RNA), like deoxyribonucleic acid (DNA), is an essential component of the cellular repair and replication process. RNA exist, within a single cell, in various structural formats which work together to translate and transfer the genetic information contained in nuclear DNA to other areas of the cell. Such translation and transfer is critical to producing the proteins that serve essential functions in the cells of the body. 

RNA interference (RNAi) is a Nobel Prize winning technology that promises to revolutionize the way drugs are discovered and developed. RNAi is a natural cellular process of gene silencing that occurs in almost all organisms. RNAi works through short double-stranded RNA (<30 base pairs) known as short interfering RNA (siRNA) which induces sequence-specific silencing of the target disease gene thus preventing disease-causing proteins from being made. However, unlike other drugs, RNA does not normally circulate and is therefore not stable in the bloodstream. This presents a major hurdle to companies currently working toward the development of RNAi therapeutics - namely, the lack of an effective delivery method for such agents. The absence of a stable, non-toxic and tissue-specific mode for systemic delivery of siRNA to the targeted (disease) cells has limited the application of this revolutionary, Nobel Prize winning technology.