Extended Data Fig. 6: rAAV9.2xstRNATyr rescued MPS-I phenotype in female mice.
From: AAV-delivered suppressor tRNA overcomes a nonsense mutation in mice
a, Workflow to assess in vivo rAAV9.2xsup-tRNATyr treatment efficacy in female IduaW401X/W401X knock-in mice (KI/KI). b, IDUA enzymatic activity in liver and heart tissue lysates derived from KI/KI mice with (+) or without (-) rAAV9.2xsup-tRNATyr treatment (n = 4 and 3, respectively). Statistical analysis was performed by two-sided Welch’s t-test. c-e, Tissue GAG (c), glucuronidase activity (d), and hexosaminidase activity (e) levels in KI/KI mice with (+) or without (-) rAAV9.2xsup-tRNATyr treatment (n = 4 or 3 per group), KI/+ mice without (-) rAAV9.2xsup-tRNATyr treatment (n = 4). Data are mean ± s.d. of individual animals (circles). Statistical analysis was performed by one-way ANOVA followed by two-sided Dunnett’s multiple comparisons test (c-e). *p < 0.05, **p < 0.01, ***p < 0.001, ns: not significant.
