Skip to main content
. 2021 Sep 23;10(10):2512. doi: 10.3390/cells10102512

Figure 2.

Figure 2

RNA-based drugs in muscular and cardiovascular pathologies. (A) “Mimics” and siRNAs act by targeting specific mRNAs to inhibit their translation. Examples include the TQJ230 siRNAs, which specifically recognize and induce the degradation of ApoA mRNA in patients with pre-existing cardiovascular diseases [91]; (B) “AntagomiRs” act by sponging endogenous miRNAs, thus preventing their translational repression. MRG-110 was used to block miR-92a activity on pro-angiogenic genes to induce wound healing [92]; (C) ASO can be used to modify the splicing of precursor mRNAs (pre-mRNA). The exon-skipping strategy applied to dystrophin exon 51 is shown as an example. In DMD patients (-ASO), genetic mutations lead to the formation of a premature stop codon (STOP symbol) in the mature transcript that causes the lack of protein translation. The use of ASO base-pairing with dystrophin exon 51 (+ASO) promotes its exclusion from the mature mRNA and leads to the translation of a shorter (but functional) protein. For each targeted exon, the ASO approved by the FDA (Food and Drug Administration) are indicated [93,94,95,96,97]; (D) VEGF modRNA used in MI patients [98]. The uridine into pseudouridine substitution is represented by the greek Ψ symbol (red). Grey line: DNA; black line: RNA; red line: Therapeutic RNA.