Extended Data Fig. 8. Off-target editing analysis in AAV9-ABE8e treated mice.
Candidate genomic loci identified by CIRCLE-seq (see Methods) were PCR-amplified and sequenced by high throughput DNA sequencing (Illumina MiSeq). Adenine base editing at the nucleotide closest to the window center was quantified by CRISPResso2 (https://github.com/pinellolab/CRISPResso2). A) Detected Off-target editing in untreated mice (n = 3) compared to mice receiving two doses of base editor AAVs sacrificed at 5 weeks of age (n = 2). B) Detected off-target editing in untreated mice (n = 3) compared to mice receiving two doses of base editor AAVs sacrificed at 30 weeks of age (n = 3). C) Assessment of RNA editing (A-to-I nucleotide changes) by ABE8e in RNA sequencing data from LV tissues of treated (n = 3) and untreated (n = 4) R403Q mice. Data are presented as mean values ±SD and significance assessed by two-sided T-test (provided in Source Data).