Fig. 1: DM1 NPC model for antisense oligonucleotide screening.

A Immunofluorescence of reprogrammed iPSC lines for the surface antigen TRA-1-60 and the nuclear pluripotency marker NANOG. B Loss of the pluripotency markers OCT4 and NANOG and expression of the neuroectodermal markers NES and PAX6. C Forebrain identity of neurons after 15 days of maturation. Patch-clamp measured D sodium currents, and E action potentials and cell-attached recordings of spontaneous cell firing activity. F Southern blot of DM1 iPSCs with a large repeat expansion in the DMPK gene. G FISH showing the presence of CUG-expanded foci in DM1 iPSCs and NPCs. H Quantification of overall CUG foci by FISH. Statistical analyses were performed using an ordinary one-way ANOVA with Tukey’s multiple comparisons test. I RT-qPCR DMPK mRNA analysis after a treatment with 500 nM IONIS 486178 ASO. A two-tailed unpaired Student’s t-test with Welch’s correction was used to determine the significance between the two groups. The error bars are presented as the mean ± SEM. SEM standard error of the mean.