(A–F) FISH detects sense RNA foci (arrows) in the cerebellum (A and B), cortex (C and D), and hippocampus (E and F) of representative 3- to 4-month-old mice. DAPI marks nuclei in blue. Scale bars: 10 μm.
(G) Distribution of RNA foci in 3- to 4- month-old (TG3C2)62 mice. Similar results are seen at 6 months.
(H) MSD immunoassay to detect poly(GP) in cortical lysates from 3- to 4-month-old mice. Error bars are SEM. n = 5–8 mice per AAV. Samples were run induplicate. ****p ≤ 0.0001 (one-way ANOVA with Tukey’s multiple comparisons test).
(I–N) IHC for poly(GP) in the cerebellum (I and J), cortex (K and L), and hippocampus (M and N) of representative 3- to 4-month-old mice. Arrows mark inclusions. Scale bars: 100 μm.
(O and P) IHC for poly(GP) in human frontal cortex of c9FTD/ALS (O) and SCA36 (P) patients. Arrows mark inclusions. Scale bars: 50 μm.
(Q and R) IHC for poly(PR) in human cerebellar granular cells of c9FTD/ALS (Q) and SCA36 (R) patients. Arrows mark inclusions. Scale bars: 25 μm.
See also Figure S4.