Fig. 4: Localized gene-editing of expanded GGC repeats reduced polyG level and rescued neuropathology in transgenic NIID mouse model. | Nature Communications

Fig. 4: Localized gene-editing of expanded GGC repeats reduced polyG level and rescued neuropathology in transgenic NIID mouse model.

From: Precise excision of expanded GGC repeats in NOTCH2NLC via CRISPR/Cas9 for treating neuronal intranuclear inclusion disease

Fig. 4: Localized gene-editing of expanded GGC repeats reduced polyG level and rescued neuropathology in transgenic NIID mouse model.

A Immunofluorescence analysis using antibody against Flag revealed a significant reduction of polyG aggregates in the striatum injected with AAV-Cas9 and AAV-sgRNA1 + 6 (treated), compared to the contralateral side injected with AAV-Cas9 and AAV-control sgRNA (untreated). Immunofluorescence analysis using antibodies against GFAP, IBA1, and NeuN demonstrated reduced GFAP (B) and IBA1 (C) expression alongside increased NeuN (D) levels in the treated striatum, compared to the contralateral untreated side. ZsGreen fluorescence (reporter encoded in the AAV-sgRNA vector) confirmed efficient AAV transduction in targeted regions. Scale bar is 200 μm and 50 μm for low- and high-magnification images, respectively. ****P < 0.0001 (polyG), ***P = 0.0001 (GFAP & IBA1), *P = 0.0245 (NeuN), two-tailed t-test. N = 3 mice per group. Data are represented as mean ± SEM. Source data are provided as a Source Data file.

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