Fig. 7: AAV stabilized in a thin film for 150 days at 4 °C performs in vivo in a dose-dependent manner equivalent to that of a frozen vector.
From: Thermostability and in vivo performance of AAV9 in a film matrix
Films prepared with high (F1S) and low (F2S) viscosity formulations were rehydrated and diluted with saline to administer 1 × 1010 (Dose 1) or 1 × 1011 (Dose 2) virus genomes of AAV9 CBA-Luc by tail vein injection. a, b Luciferase transgene expression in target organs 30 days after administration assessed by bioluminescence imaging. Mean (±standard error of the mean) values for total flux for each organ are shown. Significant differences between animals treated with each dose of the standard control formulation (FF) and the equivalent dose of FS1 and FS2 formulations were evaluated by one-way ANOVA with Dunnett’s multiple comparison tests. c Genome copies of AAV9 in organs with high transgene expression as determined by quantitative real-time PCR. Data reflect the mean (±standard error of the mean) values for each treatment group. Significant differences between subgroups with respect to dose were evaluated by one-way ANOVA with Tukey’s multiple comparison tests. a, b Diaph diaphragm, Spinal spinal cord, Gastroc gastrocnemius muscle *p < 0.05, **p < 0.01.
