Fig. 4: CRISPR-CasRx reduces G4C2 sense RNA in vivo in 149 R mice. | Nature Communications

Fig. 4: CRISPR-CasRx reduces G4C2 sense RNA in vivo in 149 R mice.

From: Dual-targeting CRISPR-CasRx reduces C9orf72 ALS/FTD sense and antisense repeat RNAs in vitro and in vivo

Fig. 4

a Schematic of experimental paradigm. CasRx PHP.eB AAV and the 149 R C9orf72 AAV9 were co-injected via intracerebroventricular (ICV) injection into P0 C57BL6/J mice. CAG-CMV enhancer/chicken β-actin promoter. CBA chicken β-actin promoter, NLS nuclear localisation signal. Created in BioRender. Cammack, A. (2024) https://BioRender.com/u85i634. b RT-qPCR of the sense repeat-containing transcripts from the 149 R AAV performed on RNA isolated from the hippocampus 3 weeks post-injection. Each data point represents one animal. RT-qPCR data presented as fold change compared to non-targeting (NT+NT) CasRx PHP.eB AAV-treated mice. n = 8 NT+NT, n = 14 10+17. c MSD immunoassay of polyGP in bulk hippocampal tissue 3 weeks post-injection. n = 7 NT+NT, n = 14 10+17. Data in (b, c) are shown as mean ± S.D, two-sided unpaired t-test. Source data are provided as a Source Data file.

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