Fig. 5: SPLICER enables efficient DNA editing and full exon skipping in a humanized mouse model of Alzheimer’s Disease.
From: SPLICER: a highly efficient base editing toolbox that enables in vivo therapeutic exon skipping
A Experimental workflow of in vivo editing following AAVrh10 injection of BEs targeting the SA and SD of APP exon 17 in the hippocampus of R1.40 mice (Created in BioRender. Miskalis, A. (2024) https://BioRender.com/f96o812). B In vivo genomic DNA editing of APP exon 17 SA and (C) SD in bulk hippocampus tissue measured using NGS. D In vivo exon skipping of APP exon 17 in bulk hippocampus tissue following treatment with BEs targeting the SA and SD. E Sashimi plot of splicing events in control mice and mice injected with BEs targeting the SA and SD of APP exon 17. For all bar graphs, values represent means and error bars indicate SD. For heatmaps, values represent means. For sashimi plots, values represent means. Each replicate value is from the hippocampus of an individual mouse. All measurements of exon skipping were performed by NGS and all DNA editing rates were measured by NGS. n = 3 for all groups; *p < 0.05; **p < 0.01; two-tailed, unpaired t-test. Source data are provided as a Source Data file.
