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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: nienhuis aw. Blood. 2002 Sep 1;100(5):1662-9. doi: 10.1182/blood-2002-02-0589. Blood. 2002. PMID: 12176886 Free article.
Current status of gene therapy for hemophilia.
Nathwani AC, Nienhuis AW, Davidoff AM. Nathwani AC, et al. Among authors: nienhuis aw. Curr Hematol Rep. 2003 Jul;2(4):319-27. Curr Hematol Rep. 2003. PMID: 12901329 Review.
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: nienhuis aw. 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: nienhuis aw. 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.
Gene Therapy for Hemophilia.
Nienhuis AW, Nathwani AC, Davidoff AM. Nienhuis AW, et al. Hum Gene Ther. 2016 Apr;27(4):305-8. doi: 10.1089/hum.2016.018. Hum Gene Ther. 2016. PMID: 27021603 Review.
Gene Therapy for Hemophilia.
Nienhuis AW, Nathwani AC, Davidoff AM. Nienhuis AW, et al. Mol Ther. 2017 May 3;25(5):1163-1167. doi: 10.1016/j.ymthe.2017.03.033. Epub 2017 Apr 11. Mol Ther. 2017. PMID: 28411016 Free PMC article. Review.
Long-term safety and efficacy following systemic administration of a self-complementary AAV vector encoding human FIX pseudotyped with serotype 5 and 8 capsid proteins.
Nathwani AC, Rosales C, McIntosh J, Rastegarlari G, Nathwani D, Raj D, Nawathe S, Waddington SN, Bronson R, Jackson S, Donahue RE, High KA, Mingozzi F, Ng CY, Zhou J, Spence Y, McCarville MB, Valentine M, Allay J, Coleman J, Sleep S, Gray JT, Nienhuis AW, Davidoff AM. Nathwani AC, et al. Among authors: nienhuis aw. Mol Ther. 2011 May;19(5):876-85. doi: 10.1038/mt.2010.274. Epub 2011 Jan 18. Mol Ther. 2011. PMID: 21245849 Free PMC article.
Our journey to successful gene therapy for hemophilia B.
Nathwani AC, Nienhuis AW, Davidoff AM. Nathwani AC, et al. Among authors: nienhuis aw. Hum Gene Ther. 2014 Nov;25(11):923-6. doi: 10.1089/hum.2014.2540. Hum Gene Ther. 2014. PMID: 25397929 Free PMC article. No abstract available.
313 results