Fig. 6: MuSK-CAART off-target effects on muscle are not observed.
a, Effects of MuSK-CAART or CART-19 (E:T 10:1) on agrin-induced AChR clustering in C2C12 mouse myotubes were visualized by α-bungarotoxin staining (×25 magnification, top row, plus bottom inset (red boxes); scale bar, 50 µm). Representative images are shown from two individual experiments. b, AChR clustering was quantitated by fluorescence intensity, measured at six different sites in each image (error bars indicate mean ± s.e.m.). One-way ANOVA with the Holm–Sidak test for multiple comparisons. c, Differentiated primary human myotubes were incubated with agrin (5 ng ml−1) or medium alone. Agrin-induced MuSK phosphorylation was confirmed by phospho-MuSK ELISA. OD450/570 value relative to medium-alone control is shown. Mann–Whitney U-test (two-tailed). d, Human myotubes were coincubated with NTD-T, MuSK-CAART or Wise-CAART in the presence or absence of agrin for 24 hours. IFNγ production was measured in cell-culture supernatants by ELISA. Error bars indicate mean ± s.d. of triplicates. One-way ANOVA with the Holm–Sidak test for multiple comparisons. NS, P > 0.05; *P < 0.05; ****P < 0.0001.
