Fig. 2

Excision of the G₄C₂ repeat expansion reverses RNA foci pathology in mutant MNs. a Schematic representation of MN generation and timings of various experiments performed. b Representative images of Isl1/2 and Tau immunostainings in 1-week-old MNs (scale bar: 50 µm). c Percentage of Isl1/2⁺ MNs from various iPSC lines (data represented as mean ± s.e.m., Con-1, N = 3; Con-2, N = 3; C9-1, N = 3; C9-1Δ, N = 3; C9-2, N = 3; C9-2Δ, N = 2; C9-3, N = 3; C9-3Δ, N = 3). d RNA fluorescent in situ hybridization showed mutant C9ORF72 MNs exhibit intra-nuclear RNA foci (scale bar: 5 µm). e Representative dot-blots for poly-GA, poly-GP, and poly-GR DPRs in mutant C9ORF72 MNs. RA retinoic acid, SAG smoothened agonist, DAPT N-[N-(3,5-Difluorophenacetyl)-L-alanyl]-S-phenylglycine t-butyl ester, BDNF Brain-derived neurotrophic factor