Science

siRNAs are short double-stranded RNAs of ~20 base pairs, made up of an antisense (AS) (or guide) strand and a sense (or passenger) strand

siRNAs utilize the endogenous RNA-induced silencing complex (RISC) to knockdown RNA targets

RISC is a multi-protein complex composed of Argonaute 2 (AGO2), trans-activation response RNA binding protein 2 and DICER1

Advances in siRNA chemistry and delivery technologies has led to clinical success of siRNA therapeutics.

At least seven siRNA therapeutics have been approved by the US Food and Drug Administration (FDA)

siRNA Design 2025