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. Author manuscript; available in PMC: 2022 Aug 10.
Published in final edited form as: Nat Biotechnol. 2022 Feb 10;40(6):938–945. doi: 10.1038/s41587-021-01171-4

Figure 4: In vivo activity of cadRNAs.

Figure 4:

(a) (i) AAV vectors used for adRNA delivery. (ii) Schematic of the in vivo experiment. (b) In vivo RNA editing efficiencies of the mPCSK9 transcript in mice livers via systemic delivery of U6 transcribed linear (U6+27) and genetically encoded circular adRNAs packaged in AAV8. Values represent mean +/− SEM (n=3; p=0.0002; unpaired t-test, two-tailed). (c) Relative expression levels of circular adRNAs. Values represent mean +/− SEM (n=3; p=0.0305; unpaired t-test, two-tailed). (d) mPCSK9 transcript levels relative to GAPDH. Values represent mean +/− SEM (n=3; p=0.6179, p=0.6125, p=0.9323; unpaired t-test, two-tailed). (e) Schematic of the IDUA-W392X mRNA, and RNA editing experiment. (f) In vivo UAG-to-UGG RNA editing efficiencies of the IDUA transcript in mice livers via systemic delivery of genetically encoded circular adRNAs packaged in AAV8. Values represent mean +/− SEM (n=3). (g) IDUA transcript levels relative to GAPDH. Values represent mean +/− SEM (n=3; p=0.1185, p=0.3815, p=0.0042; unpaired t-test, two-tailed). (h) GAG content in mice livers of AAV8-scrambled.2x.circular.200.100 and AAV8-IDUA.2x.circular.200.100 injected IDUA-W392X mice. Wild type C57BL/6J mice were included as controls. Values represent mean +/− SEM (n=3; p=0.0285; unpaired t-test, two-tailed).