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Reversal of C9orf72 mutation-induced transcriptional dysregulation and pathology in cultured human neurons by allele-specific excision.
Sachdev A, Gill K, Sckaff M, Birk AM, Aladesuyi Arogundade O, Brown KA, Chouhan RS, Issagholian-Lewin PO, Patel E, Watry HL, Bernardi MT, Keough KC, Tsai YC, Smith AST, Conklin BR, Clelland CD. Sachdev A, et al. Proc Natl Acad Sci U S A. 2024 Apr 23;121(17):e2307814121. doi: 10.1073/pnas.2307814121. Epub 2024 Apr 15. Proc Natl Acad Sci U S A. 2024. PMID: 38621131 Free PMC article.