Figure 6.

Summary of editing outcomes in the brain of SCA2 transgenic mice

(A) Editing outcomes from nCATS (Figures 2, 3, 4, and 5) or from ddPCR (Figures 2I–2L; indels). (B) Frequency of integrations that were fragments or full-length AAVs. (C) Data observed in SCA2 transgenic mice brain offer predictions for expected editing outcomes in normal and SCA2 humans. SCA2 transgenic mice with multiple copies have multiple target sites (red arrowheads) on a single chromosome compared to humans with targets sites on two chromosomes. The asterisk denotes the large deletions and transgene rearrangements unique to editing in a transgenic mouse model.