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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: mcintosh j. 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.
An update on gene therapy for hemophilia.
Nathwani AC, McIntosh J, Davidoff AM. Nathwani AC, et al. Among authors: mcintosh j. Curr Hematol Rep. 2005 Jul;4(4):287-93. Curr Hematol Rep. 2005. PMID: 16009043
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: mcintosh j. 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: mcintosh j. 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.
Enhancing transduction of the liver by adeno-associated viral vectors.
Nathwani AC, Cochrane M, McIntosh J, Ng CY, Zhou J, Gray JT, Davidoff AM. Nathwani AC, et al. Among authors: mcintosh j. Gene Ther. 2009 Jan;16(1):60-9. doi: 10.1038/gt.2008.137. Epub 2008 Aug 14. Gene Ther. 2009. PMID: 18701909 Free PMC article.
1,271 results