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Correction of DMD exon 45 deletion mutation by NHBEJ editing
(A) Schematic illustration of the DMD exon 45 deletion locus, transcription, and CRISPR NHBEJ targeting. NMD, nonsense-mediated decay. (B) PCR-genotyping results from a pool of DMD ex45del fibroblasts edited with SpCas9 protein and either the pair 1 (T1 + T2) or pair 2 (T3 + T4) gRNAs. Controls were transfected with SpCas9 only (n = 3). (C) Semi-quantification of NHBEJ frequency based on Sanger sequencing and ICE analysis.
