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Biodistribution and safety of a single rAAV3B-AAT vector for silencing and replacement of alpha-1 antitrypsin in Cynomolgus macaques.
Blackwood M, Gruntman AM, Tang Q, Pires-Ferreira D, Reil D, Kondratov O, Marsic D, Zolotukhin S, Gernoux G, Keeler AM, Mueller C, Flotte TR. Blackwood M, et al. Mol Ther Methods Clin Dev. 2024 Jan 30;32(1):101200. doi: 10.1016/j.omtm.2024.101200. eCollection 2024 Mar 14. Mol Ther Methods Clin Dev. 2024. PMID: 38445045 Free PMC article.
Death after High-Dose rAAV9 Gene Therapy in a Patient with Duchenne's Muscular Dystrophy.
Lek A, Wong B, Keeler A, Blackwood M, Ma K, Huang S, Sylvia K, Batista AR, Artinian R, Kokoski D, Parajuli S, Putra J, Carreon CK, Lidov H, Woodman K, Pajusalu S, Spinazzola JM, Gallagher T, LaRovere J, Balderson D, Black L, Sutton K, Horgan R, Lek M, Flotte T. Lek A, et al. Among authors: blackwood m. N Engl J Med. 2023 Sep 28;389(13):1203-1210. doi: 10.1056/NEJMoa2307798. N Engl J Med. 2023. PMID: 37754285
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. Among authors: blackwood mt. Nat Commun. 2022 Oct 21;13(1):6286. doi: 10.1038/s41467-022-33332-7. Nat Commun. 2022. PMID: 36271076 Free PMC article.
65 results