Extended Data Fig. 8: Durable efficacy in IduaKI/KI mice at 20 weeks post-treatment.
From: An engineered UGA suppressor tRNA gene for disease-agnostic AAV delivery
(a) Workflow of in vivo assessment of the therapeutic efficacy of MyoAAV.2xCuO-hNFS-R8(VG4) in IduaKI/KI mice at 20 weeks post-treatment. (b) Bar graph showing the IDUA enzymatic activities in the indicated tissues collected from untreated wild-type mice (WT), untreated IduaKI/KI mice, or IduaKI/KI mice treated with MyoAAV.2xCuO-hNFSR8(VG4) and sacrificed at 20 weeks post-treatment. Data are normalized to the WT levels as 100%. (c) Bar graph showing the ratios of mouse Idua mRNA to Tbp mRNA (mIdua/mTbp) in the indicated tissues collected from untreated wild-type mice (WT), untreated IduaKI/KI mice, or IduaKI/KI mice treated with MyoAAV.2xCuOhNFS-R8(VG4). In b and c, each dot represents an individual animal (n = 6). Data are presented as mean and standard deviation. Statistical analysis was performed by unpaired two-sided t-test in b, or one-way ANOVA followed by Dunnett’s multiple comparisons test in c. Schematic in a created in BioRender. Zou, H. (2025) https://BioRender.com/dqjni51.
