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2020 12
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CRISPR/Cas9-mediated excision of ALS/FTD-causing hexanucleotide repeat expansion in C9ORF72 rescues major disease mechanisms in vivo and in vitro.
Meijboom KE, Abdallah A, Fordham NP, Nagase H, Rodriguez T, Kraus C, Gendron TF, Krishnan G, Esanov R, Andrade NS, Rybin MJ, Ramic M, Stephens ZD, Edraki A, Blackwood MT, Kahriman A, Henninger N, Kocher JA, Benatar M, Brodsky MH, Petrucelli L, Gao FB, Sontheimer EJ, Brown RH, Zeier Z, Mueller C. Meijboom KE, et al. Nat Commun. 2022 Oct 21;13(1):6286. doi: 10.1038/s41467-022-33332-7. Nat Commun. 2022. PMID: 36271076 Free PMC article.
Comprehensive evaluation of human-derived anti-poly-GA antibodies in cellular and animal models of C9orf72 disease.
Jambeau M, Meyer KD, Hruska-Plochan M, Tabet R, Lee CZ, Ray-Soni A, Aguilar C, Savage K, Mishra N, Cavegn N, Borter P, Lin CC, Jansen-West KR, Jiang J, Freyermuth F, Li N, De Rossi P, Pérez-Berlanga M, Jiang X, Daughrity LM, Pereira J, Narayanan S, Gu Y, Dhokai S, Dalkilic-Liddle I, Maniecka Z, Weber J, Workman M, McAlonis-Downes M, Berezovski E, Zhang YJ, Berry J, Wainger BJ, Kankel MW, Rushe M, Hock C, Nitsch RM, Cleveland DW, Petrucelli L, Gendron TF, Montrasio F, Grimm J, Polymenidou M, Lagier-Tourenne C. Jambeau M, et al. Proc Natl Acad Sci U S A. 2022 Dec 6;119(49):e2123487119. doi: 10.1073/pnas.2123487119. Epub 2022 Dec 1. Proc Natl Acad Sci U S A. 2022. PMID: 36454749 Free PMC article.
51 results