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Page 1
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.
Adenovirus-associated virus vector-mediated gene transfer in hemophilia B.
Nathwani AC, Tuddenham EG, Rangarajan S, Rosales C, McIntosh J, Linch DC, Chowdary P, Riddell A, Pie AJ, Harrington C, O'Beirne J, Smith K, Pasi J, Glader B, Rustagi P, Ng CY, Kay MA, Zhou J, Spence Y, Morton CL, Allay J, Coleman J, Sleep S, Cunningham JM, Srivastava D, Basner-Tschakarjan E, Mingozzi F, High KA, Gray JT, Reiss UM, Nienhuis AW, Davidoff AM. Nathwani AC, et al. Among authors: ng cy. N Engl J Med. 2011 Dec 22;365(25):2357-65. doi: 10.1056/NEJMoa1108046. Epub 2011 Dec 10. N Engl J Med. 2011. PMID: 22149959 Free PMC article. Clinical Trial.
Therapeutic levels of FVIII following a single peripheral vein administration of rAAV vector encoding a novel human factor VIII variant.
McIntosh J, Lenting PJ, Rosales C, Lee D, Rabbanian S, Raj D, Patel N, Tuddenham EG, Christophe OD, McVey JH, Waddington S, Nienhuis AW, Gray JT, Fagone P, Mingozzi F, Zhou SZ, High KA, Cancio M, Ng CY, Zhou J, Morton CL, Davidoff AM, Nathwani AC. McIntosh J, et al. Among authors: ng cy. Blood. 2013 Apr 25;121(17):3335-44. doi: 10.1182/blood-2012-10-462200. Epub 2013 Feb 20. Blood. 2013. PMID: 23426947 Free PMC article.
515 results