Figure 2. mxABE-mediated correction of mutant DMD RNA.

(A) The reporter construct containing the mCherry cassette fused with a 2A peptide, mutant human exon 30 (c.4174C>T), and ATG-removed GFP. Correction of the stop codon within the target sequence allows GFP expression. (B) Flow cytometry analysis of GFP expression in HEK293T cells transfected with 24 gRNAs. (C) Deep sequencing of the reporter RNA transcribed from the reporter vector after GFP rescue experiment. (D and E) Comparison of the editing efficiencies of different mxABE vectors by flow cytometry (D) and deep sequencing (E). (F) Measurement of bystander A-to-I editing rate for multiple adenosines within a 50 nt region of the DMD^E30mut target sequence. gRNA g6 was used in the analysis. Adenosines (A) with position number are indicated from the 5′ to the 3′ end in the 50 nt target sequence. Data are represented as mean ± SEM (n = 3). **P < 0.01 using unpaired 2-tailed Student’s t test.