Fig. 3: Evaluating the impact of WPRE on transgene expression mediated by Anc80L65.
From: Optimized in vivo base editing restores auditory function in a DFNA15 mouse model
A Whole-mount preparations of the cochlea showing the distribution of EGFP fluorescent protein in hair cells after AAV injection into the P1–3 mouse cochlea, n = 4 independent experiments with similar results. Myo7a (purple): hair cell marker. Scale bar, 50 μm. B The number of EGFP-positive OHCs (left) and IHCs (right) per 100 μm length, n = 4. WT vs Pou4f3WT/Q113*: in OHCs, p = 0.000034 (apex), p < 0.000001(middle), p = 0.000061 (base), in IHCs, p = 0.000019 (apex), p = 0.000012 (middle), p = 0.000049 (base). C, D Split-site screening of split-SchABE8e. C Western blotting results of the intein-mediated recombination expression of the five split-SchABE8e variants. D Quantitative analysis of SchABE8e expression by calculating the grayscale intensity of the HA tag from three independent experiments using ImageJ software (Fiji, Inc.). E Heatmap of editing efficiency of the split-SchABE8e variants with sgRNA3. Data were collected from three independent experiments. Data in (B, D) are represented as mean ± SEM. Statistical significance was determined by unpaired two-tailed Student’s t-tests (***p < 0.001). Source data are provided as a Source data file.
