Fig. 6: Disease phenotype amelioration in CRISPR/Cas9 edited FRDA-LD DRGOs. | Nature Communications

Fig. 6: Disease phenotype amelioration in CRISPR/Cas9 edited FRDA-LD DRGOs.

From: Frataxin gene editing rescues Friedreich’s ataxia pathology in dorsal root ganglia organoid-derived sensory neurons

Fig. 6

a, d Representative images of FRDA patient line derived DRGOs along with short and long deletion isogenic lines, respectively, as compared with a control (CTRL) healthy donor derived DRGO stained for NF200 (a) and in BF (b), and neuritis area quantification for PTL6 and its isogenic lines (c) and PTS36 and its isogenic lines (d). Mean ± s.d., n = 4 independent experiments 3 organoids/line/experiment. *P < 0.05; **P < 0.01; ***P < 0.001; one-way ANOVA with Bonferroni correction. Scale bar: 500 μm. e–j Quantitative analysis of transcriptional levels of PVALB (propriorecetors) (e, f), NTRK1 (nociceptors) (g, h) and CACNA1H (mechanoreceptors) (i, j) in control, FRDA patients and their isogenic lines. Expression levels are normalized to actin. Mean ± s.d., n = 3 independent experiments, 8–12 organoids/line/experiment. *P < 0.05; **P < 0.01; ***P < 0.001; one-way ANOVA with Bonferroni correction.

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