Figure 3 | Scientific Reports

Figure 3

From: Spinal muscular atrophy phenotype is ameliorated in human motor neurons by SMN increase via different novel RNA therapeutic approaches

Figure 3

MO-10-34 ameliorates the SMA phenotype in iPSC-MNs, increasing cell survival in long-term cultures.

(A) Schematic differentiation protocol detailing the differentiation of iPSCs into motor neurons (MNs). (B) iPSCs (wild-type (WT), treated (TR)-SMA iPSCs and scramble SMA-treated iPSCs) differentiated into MNs and expressed typical MN markers: SMI32-positive (green) and ChAT-positive (red). MNs appear yellow, representing the merging of red and green colors. Nuclei are labeled with DAPI (blue). SMA-MNs derived from scramble SMA-treated iPSCs appeared smaller and more sparse than MNs derived from WT-iPSCs. Notably, MNs derived from MO-treated iPSCs presented a rescue of cell density and size in culture, similar to MNs derived from WT iPSCs. (C) Quantification of MNs at week 10 after differentiation from iPSCs showed increased MN survival of TR SMA iPSC–derived MNs compared with scramble SMA iPSC–derived MNs (*p < 0.001, ANOVA). Values represent means ± s.e.m. from five independent experiments performed in triplicate. (D) MO-treated iPSC-derived MNs presented a significantly increased number of gems relative to MNs derived from scramble-treated SMA iPSCs. Gem counts in wild-type (WT) and heterozygous (HET) cells are shown for comparison (*p < 0.01, ANOVA). Values represent means ± s.e.m. from five independent experiments performed in triplicate. Scale bar: 75 μm.

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