Figure 1.

INTASYLs are asymmetric RNAi compounds comprised of a small duplex region (<15 base pairs) and a single-stranded phosphorothioate tail (≥ 6 nucleotides). In addition, INTASYL compounds are chemically modified with stabilizing and hydrophobic modifications (eg sterol), which confer stability, efficient cellular uptake and reduced inflammatory response (A). INTASYL functions through the RNAi pathway, a naturally occurring cellular process. Introduction of short double stranded RNA, such as INTASYL, into cells can result in association with the RNA induced silencing complex (RISC) to target and degrade complementary mRNA sequences and silence target genes (B).