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In C9orf72 ALS/FTD, ADAR2 was found to be mislocalized in postmortem spinal cord tissue from C9orf72 patients, hiPSC MNs derived from C9orf72 patients, and in 6-month-old mice expressing the hexanucleotide repeat expansion via P0 intracerebroventricular (ICV) injection with AAV-(G4C2)149 [56]

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[DOI] [PMC free article] [PubMed] [Google Scholar] 56.Finkelstein JD