Fig. 4
scAAV2-DX2-miR142-3p target sequence selectively expressed in the target organ A Comparison of transgene expression between scAAV-DX2 and scAAV2-DX2-miR142-3p target sequence plasmid in hematopoietic cells and neuronal cells. B Relative expression ratio of DX2 with or without miR142 inhibitor in THP-1 cells. ‘ + ’ means 10 pmol of miR142 inhibitor. C Body distribution test for scAAV-DX2 in intracranially injected mice. D–G Assessing scAAV-DX2 spreading and expression during intrathecal injection administration between lumbar and blood. D DNA level of DX2 in low-dose injected mice E DX2 level of DX2 in high-dose injected mice. F RNA level of DX2 in low-dose injected mice G RNA level of DX2 in high-dose injected mice. animal number: n = 6, Low dose: 2.4 × 1010vg/animal, High dose: 9.6 × 1010vg/animal. ns: no-significant; *: p < 0.05; **:p < 0.01; ***: p < 0.001 TS miR142-3p target sequence, SC spinal cord, CB celebellum, CL cerebral lobes, BS Brain stem, WB whole blood