Fig. 2: Neurodevelopmental disease signatures of SMA SCOs. | Nature Communications

Fig. 2: Neurodevelopmental disease signatures of SMA SCOs.

From: Targeted antisense oligonucleotide treatment rescues developmental alterations in spinal muscular atrophy organoids

Fig. 2: Neurodevelopmental disease signatures of SMA SCOs.The alternative text for this image may have been generated using AI.

a, b Representative images of SOX2 (green) and TUJ1 (red) staining at 1 and 2 mo in CTRL and SMA SCOs. Scale bar: 200 µm. Nuclei were stained with DAPI (blue). c Quantification of SOX2-positive cells (as a percentage of cells revealed by DAPI staining) at 1 (circles) and 2 mo (triangles) (two-way ANOVA, Sidak’s multiple comparisons test: p < 0.0001 and p = 0.0148 for CTRL and SMA, respectively. n = 28 from D30 and D60 CTRL SCOs; n = 4 C1, n = 4 C2, n = 3 C4, n = 3 C5; n = 8 C1, n = 3 C4, n = 3 C5; n = 33 from D30 and D60 SMA SCOs n = 4 S1, n = 7 S2, n = 6 S4, n = 2 S5; n = 5 S2, n = 4 S3, n = 5 S4). Box plots show the minimum and maximum value in the series along with median and 25–75 percentile. d Expression of TUJ1 increase over time in the CTRL, meanwhile in the SMA SCOs there is a trend of decreasing in the expression of TUJ1 (two-way ANOVA, Sidak’s multiple comparisons test: p = 0.0002 and p = 0.0764 for CTRL and SMA, respectively. n = 33 from D30 and D60 CTRL SCOs n = 4 C1, n = 5 C2, n = 3 C3, n = 3 C4, n = 3 C5; n = 4 C1, n = 3 C2, n = 3 C3, n = 3 C4, n = 2 C5; n = 33 from D30 and D60 SMA SCOs n = 4 S1, n = 2 S2, n = 4 S3, n = 6 S4, n = 2 S5; n = 4 S1, n = 3 S2, n = 3 S3, n = 5 S4). e Stacked bar plot showing the ratio of SOX2 and TUJ1 at 1 mo and 2 mo in CTRL and SMA SCOs. (Mann–Whitney test: p = 0.0444 and p = 0.1734 CTRL vs. SMA at 1 mo and 2 mo, respectively. n = 24 from D30 and D60 CTRL SCOs n = 4 C1, n = 4 C2, n = 3 C4, n = 3 C5; n = 5 C1, n = 3 C4, n = 2 C5; n = 25 from 1 mo and 2 mo SMA SCOs n = 4 S1, n = 2 S2, n = 6 S4, n = 2 S5; n = 3 S2, n = 3 S3, n = 5 S4). f UMAP plot showing cells colored by pseudotime score, with label annotation of major cell types. g Left: Density plots of pseudotime score distributions in CTRL (blue) and SMA (gray) cells (two-sided Kolmogorov–Smirnov (KS) test, p = 2.16e–08). Right: Cumulative distribution plot of pseudotime scores in CTRL and SMA cells. h Density plots of pseudotime scores for individual cell types, comparing CTRL (blue) and SMA (gray). Significant differences in pseudotime distributions were assessed using the two-sided KS test. Asterisks indicate significance levels (*p < 0.05, **p < 0.01, ***p < 0.001, ****p < 0.0001). Source data are provided as a Source Data file.

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