Fig. 6: Therapeutic dual AAV-SchABE8e-sgRNA3 is safe in the inner ear.
From: Optimized in vivo base editing restores auditory function in a DFNA15 mouse model
A Click ABR and TB-ABR thresholds at 1-, 2-, and 3-month post AAV injection. Neonatal WT mice (red, n = 3) were injected with the same dose of dual AAV-SchABE8e-sgRNA3 into the inner ear in the pharmacodynamic assay. Click ABR and TB-ABR were performed at 1-, 2-, and 3-month post-injection. B Representative immunofluorescence images of cochleae from WT and WT mice injected with dual AAV-SchABE8e-sgRNA3 at P90, n = 4 independent experiments with similar results. Myo7a (magenta): hair cell marker. Scale bar, 20 μm. C Cell counts comparison of OHCs and IHCs corresponding to (B), n = 4. D Behavior tests comparison between WT (black) and WT mice injected with dual AAV-SchABE8e-sgRNA3 (red) at P90. Left, n = 7, right, n = 6. E Body weight comparison between WT (black) and WT mice injected with dual AAV-SchABE8e-sgRNA3 (red), n = 6. Data in (A, C, D, E) are represented as mean ± SEM. Statistical significance was determined by two-tailed Student’s t-tests (ns no significant difference). Source data are provided as a Source data file.
