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. 2022 Nov 21;119(48):e2210532119. doi: 10.1073/pnas.2210532119

Fig. 2.

Fig. 2.

Compound 1 selectively alleviates disease-associated defects in patient-derived cells. (A) Effect of 1 on poly(GP) abundance in protein lysate extracted from c9ALS patient-derived LCLs (n = 3 C9orf72 LCLs, three replicates per concentration in each line). (B) Effect of 1 on poly(GP) abundance in protein lysate extracted from c9ALS patient-derived iPSCs (n = 4 C9orf72 iPSC lines, three replicates per concentration in each line). (C) Effect of 1 on poly(GP) abundance in protein lysate extracted from patient-derived iPSNs (n = 3 C9orf72 iPSN lines, three replicates per concentration in each line). (D) Effect of 1 on C9orf72 intron 1 abundance, which harbors r(G4C2)exp, in c9ALS patient-derived LCLs, as determined by qRT-PCR with intron 1-specific primers (n = 3 C9orf72 LCLs, three replicates per concentration in each line). (E) Effect of 1 on C9orf72 intron 1 abundance in c9ALS patient-derived iPSCs, as determined by qRT-PCR with intron 1-specific primers (n = 4 C9orf72 iPSC lines, three replicates per concentration in each line). (F) Effect of 1 on C9orf72 intron 1 abundance in c9ALS patient-derived iPSNs, as determined by qRT-PCR with intron 1-specific primers primers (n = 3 C9orf72 iPSN lines, three replicates per concentration in each line). (G) Effect of 1 on C9orf72 intron 1 3′ splice in a c9ALS patient-derived LCL, as determined by qRT-PCR with primers spanning the intron 1–exon 2 junction (n = 1 C9orf72 LCL, three replicates per concentration). (H) Effect of 1 on C9orf72 intron 1 3′ splice in a c9ALS patient-derived iPSC line, as determined by qRT-PCR with primers spanning the intron 1–exon 2 junction (n = 1 C9orf72 iPSC line, three replicates per concentration). (I) Effect of 1 on C9orf72 intron 1 3′ splice in a c9ALS patient-derived iPSN, as determined by RT-qPCR with primers spanning the intron 1–exon 2 junction (n = 1 C9orf72 iPSN line, three replicates per concentration). RNA quantification was measured relative to GAPDH. Vehicle indicates 0.1% (v/v) dimethyl sulfoxide (DMSO). *P < 0.05, **P < 0.01, ***P < 0.001 , ****P < 0.0001, as determined by a One Way ANOVA with multiple comparisons. Error bars are reported as SD.