Fig. 1: Assessment of on-target and off-target editing by candidate clinical lead gRNAs.
From: Improved specificity and efficiency of in vivo adenine base editing therapies with hybrid guide RNAs
a, Schematics of the genomic sites of the five loci that are evaluated in this study, adapted from the UCSC Genome Browser (GRCh38/hg38). Each vertical yellow bar indicates either the G altered to A (in red) by a pathogenic variant, or the target A (in red) at a splice site intended for disruption. Each horizontal grey bar indicates the protospacer (thick) and protospacer-adjacent motif (PAM) (thin) sequences targeted by the respective gRNA. b, Schematic of the ONE-seq methodology.
