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. 2020 Apr 16;28(7):1696–1705. doi: 10.1016/j.ymthe.2020.04.009

Figure 6.

Figure 6

Feasibility Evaluation of ABE Editing to Correct Nonsense Mutations Causing Dysferlinopathy

(A) The target sequences and gRNAs of three nonsense mutations causing dysferlinopathy. The mutations are shown in red, and the PAM sequences are shown in blue. (B) Representative merged fluorescence and bright-field images of HEK293 cells transfected with R377X and Q1278X reporters plus or minus their corresponding gRNAs and ABEmax. Scale bar: 100 μm. (C) Representative merged fluorescence and bright-field images of HEK293 cells transfected with the Q605X reporter, Q605X gRNA and ABEmax, xABE, or ABE-NG. Scale bar: 100 μm. (D) Quantification of GFP+ cells for the conditions described in (B). (E) Quantification of GFP+ cells for the conditions described in (C). ∗∗∗p < 0.001; ∗∗∗∗p < 0.0001.