Glossary
Small interfering RNA (siRNA)
A short double stranded RNA that silences a gene through the RNA interference pathway.
A small interfering RNA is a short double stranded RNA, typically about twenty one base pairs, that triggers RNA interference. Its guide strand is loaded into the RISC complex, which then finds and cuts messenger RNA matching the guide sequence.
Because RISC acts catalytically, siRNA drugs can be highly potent and long acting, especially when conjugated to GalNAc for delivery to the liver.
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