Figure 1.

The RNAi mechanism and entry points of RNAi therapeutics

The left-hand side shows the RNAi pathway and formation of siRNAs. The right-hand side shows different entry points for DNA-based or RNA-based RNAi therapeutics that enters the cell and are loaded into RISC to mediate homology-dependent degradation of target mRNA. Delivery platforms for DNA-based therapeutics include viral and non-viral vectors encoding Pol II driven miRNA mimics or Pol III-driven shRNA. RNAi-based therapeutics are delivered naked as GalNAc-conjugated, modified, or unmodified Dicer-substrate siRNA or synthetic siRNAs or complexed in nanoparticles (gold, lipid, or polymer based). Created using BioRender.com.