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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.
In vivo lentiviral vector gene therapy to cure hereditary tyrosinemia type 1 and prevent development of precancerous and cancerous lesions.
Nicolas CT, VanLith CJ, Hickey RD, Du Z, Hillin LG, Guthman RM, Cao WJ, Haugo B, Lillegard A, Roy D, Bhagwate A, O'Brien D, Kocher JP, Kaiser RA, Russell SJ, Lillegard JB. Nicolas CT, et al. Among authors: kocher jp. Nat Commun. 2022 Aug 25;13(1):5012. doi: 10.1038/s41467-022-32576-7. Nat Commun. 2022. PMID: 36008405 Free PMC article.
163 results