Fig. 1: In vitro studies of mdx4cv mutation correction using ABE-NG.
From: Efficient precise in vivo base editing in adult dystrophic mice
a Genomic DNA, encoded amino acids and guide RNA with PAM (highlighted in blue) sequences at the stop codon mutation site (red). b The reporter construct contains a puromycin resistance cassette fused with E2A peptide, mdx4cv target sequence and ATG-removed EGFP. Correction of the stop codon within the target sequence would allow EGFP expression. c Fluorescence microscopy images of HEK293 cells transfected with reporter alone, or reporter, gRNA and one of the base editors (ABEmax, ABE-x, and ABE-NG). Scale bar: 500 µm. d–e Flow cytometry analysis of EGFP expression in HEK293 cells transfected as described in (c). n = 3 wells/group; one-way ANOVA with Turkey’s multiple comparisons test. Data are mean ± SD.
