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Fig. 1
HNRNPK prevents the repeat RNA-induced axonopathy in a transient zebrafish model specific for C9orf72 RNA toxicity. a Illustration of the C9orf72 RNA toxicity zebrafish model. b, c SV2 immunostaining of motor axons in 30 hpf zebrafish embryos injected with GFP control mRNA (b) and 91 C9orf72 hexanucleotide repeats in sense direction (91S) (c). Scale bar = 50 µm. d, e Percentage of axonal length compared to control (d) and percentage of embryos with abnormal branching (e) in 30 hpf zebrafish embryos injected with GFP control and 91S repeat RNA at equimolar dose (0.844 µM) (N = 4 experiments). Data represent mean ± SEM. Statistical significance was evaluated with unpaired t test; *P < 0.05, ****P < 0.0001. f, g Effect of HNRNPK mRNA injection (0.071-0.142-0.284-0.568 µM) on the 91S repeat RNA-induced axonopathy showing a dose-dependent rescue of axonal length (f) and abnormal branching (g) (N = 3 experiments). Data represent mean ± SEM. Statistical significance was evaluated with one-way ANOVA and Tukey’s multiple comparison test; *P < 0.05, **P < 0.01, ****P < 0.0001