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Sustained high-level expression of human factor IX (hFIX) after liver-targeted delivery of recombinant adeno-associated virus encoding the hFIX gene in rhesus macaques.
Nathwani AC, Davidoff AM, Hanawa H, Hu Y, Hoffer FA, Nikanorov A, Slaughter C, Ng CY, Zhou J, Lozier JN, Mandrell TD, Vanin EF, Nienhuis AW. Nathwani AC, et al. Among authors: ng cy. Blood. 2002 Sep 1;100(5):1662-9. doi: 10.1182/blood-2002-02-0589. Blood. 2002. PMID: 12176886 Free article.
Self-complementary adeno-associated virus vectors containing a novel liver-specific human factor IX expression cassette enable highly efficient transduction of murine and nonhuman primate liver.
Nathwani AC, Gray JT, Ng CY, Zhou J, Spence Y, Waddington SN, Tuddenham EG, Kemball-Cook G, McIntosh J, Boon-Spijker M, Mertens K, Davidoff AM. Nathwani AC, et al. Among authors: ng cy. Blood. 2006 Apr 1;107(7):2653-61. doi: 10.1182/blood-2005-10-4035. Epub 2005 Dec 1. Blood. 2006. PMID: 16322469 Free PMC article.
Safe and efficient transduction of the liver after peripheral vein infusion of self-complementary AAV vector results in stable therapeutic expression of human FIX in nonhuman primates.
Nathwani AC, Gray JT, McIntosh J, Ng CY, Zhou J, Spence Y, Cochrane M, Gray E, Tuddenham EG, Davidoff AM. Nathwani AC, et al. Among authors: ng cy. Blood. 2007 Feb 15;109(4):1414-21. doi: 10.1182/blood-2006-03-010181. Epub 2006 Nov 7. Blood. 2007. PMID: 17090654 Free PMC article.
6,105 results