Fig. 2: Base editing successfully reverts classical FA phenotypes. | Nature Communications

Fig. 2: Base editing successfully reverts classical FA phenotypes.

From: Adenine base editing efficiently restores the function of Fanconi anemia hematopoietic stem and progenitor cells

Fig. 2

a Schematics of experimental design to edit FA-55 or FA-75 LCLs. Cells were edited with base editor mRNA and synthetic gRNA and grown for 30 days in culture. Editing efficiencies were assessed by amplicon NGS, as shown in Fig. 1c. (Created with BioRender.com). b MMC resistance of edited FA-A LCLs. Black line indicates healthy donor (HD) LCL response to increasing doses of MMC. Dashed blue or green lines represent FA-55 or FA-75 LCLs, respectively. Solid blue or green lines represent FA-55 or FA-75 edited pools, respectively. The graphs summarize the mean of 3 biological replicates, error bars indicate SD. c Representative Western blots. Indicated cell populations were challenged with 1 μM MMC for 24 h. Protein extracts were analyzed by Western blot with indicated antibodies (anti-FANCA, anti-FANCD2, anti-HSP60). FANCD2 or FANCD2-Ub bands are indicated by arrows. (n = 2, biologically independent experiments). Source data are provided as a Source Data file.

Back to article page