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. 2020 Jan 14;28(4):1177–1189. doi: 10.1016/j.ymthe.2020.01.005

Figure 1.

Figure 1

CBE-Mediated Base Editing Reduces SOD1 Gene Expression

(A) Schematic of the human SOD1 gene and the CBE 4 target site. Arrowhead indicates the target cytosine within the Gln codon. Unpaired bases in the target site indicate the protospacer. TSS, transcriptional start site. (B) Human SOD1 mRNA in HEK293T cells by quantitative qRT-PCR 12 days after transfection with CBEs. Data are normalized to a non-targeted CBE (n = 3). (C) (Top) Western blot of human SOD1 protein in cell lysate from HEK293T cells 12 days after transfection with CBE 4. (Bottom) Quantitation of western blot. SOD1 protein in each lane was normalized to GAPDH protein. Values were normalized to a non-targeted CBE (n = 3). (D) Plot showing the mean frequency change of the target cytosine in the SOD1 gene by deep sequencing in HEK293T cells 6 days after transfection with CBE 4 or a non-targeted control (NTG), n = 3. Error bars indicate SD. *p < 0.05, **p < 0.01, ***p < 0.001; (B and C) two-tailed unpaired t test; (D) one-tailed unpaired t test.