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. 2021 Mar 18;18(4):1491–1506. doi: 10.1021/acs.molpharmaceut.0c01238

Figure 1.

Figure 1

RNAi-based therapeutic main approaches: single-stranded antisense oligonucleotides (ASOs) and double-stranded small interfering RNA (siRNA). ASOs: Once bound to the target mRNA, ASOs can form an RNA–DNA hybrid that becomes a substrate for RNase H, which results in mRNA degradation. siRNA: Double-stranded RNA (dsRNA) is processed by Dicer into siRNA. The guide RNA strand is incorporated into the RNA-induced silencing complex (RISC) and Argonaute 2 (Ago2). Ago2 cleaves the passenger strand, and siRNA/RISC complex then binds the complementary sequence of the target mRNA resulting in the degradation of the target transcript.