Extended Data Fig. 6. FUS silencing by ION363 in FUS-ALS mice.
(a) Representative immunostaining of lumbar spinal cord sections from NTC-treated animals with an anti-ASO antibody. The staining shows widespread nuclear and cytoplasmic distribution at 1 and 4 months of age that disappears at 6 months. Scale bar=100 µm. (b) Representative immunostaining of lumbar spinal cord sections from FUS ASO-treated animals with an anti-ASO antibody. The staining shows widespread nuclear distribution at 1 and 4 months of age that disappears at 6 months. Scale bar=100 µm. (c) Immunoblot of lumbar spinal cord protein from 6-month-old WT/WT and P517L/WT animals treated with non-targeted control (NTC) or anti-FUS oligonucleotide (ION363). Each lane represents a separate animal. (d) Quantitation of FUS protein levels in lumbar spinal cord lysates from 6-month-old animals shown in (c). One-way ANOVA was performed to assess the differences in FUS levels. N = 3 animals per group.