Fig. 2: Effects of shRNA, NAGK-DS, and of NAGKD107A on Q74 aggregate formation.

A The HEK293T cellular model. HEK293T cells were transfected with indicated plasmids, double-labeled with indicated antibodies, and then stained with DAPI as described in Fig. 1. (a) Typical epifluorescence images. (b, c) Cells (n = 320) with aggregates were counted; results are expressed as percentages of all cells (b). Aggregates were classified as small (≤0.8 µm), medium (0.8–1.5 µm), or large (≥1.5 µm) and percentages in each category are shown (c). B The neuronal model. (a) Typical epifluorescence images. (b) Cells containing Q74 aggregates were counted; results are expressed as percentages of total cell counts (n = 100). Annotations are same as those used in Fig. 1. ***p < 0.001. Scale bar = 10 µm.