Our journey to successful gene therapy for hemophilia B.
作者信息
Nathwani Amit C, Nienhuis Arthur W, Davidoff Andrew M
机构信息
1 Katharine Dormandy Haemophilia Centre and Thrombosis Unit , Royal Free NHS Foundation Trust, London NW3 2QG, United Kingdom .
出版信息
Hum Gene Ther. 2014 Nov;25(11):923-6. doi: 10.1089/hum.2014.2540.
Abstract
摘要
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Therapeutic levels of FVIII following a single peripheral vein administration of rAAV vector encoding a novel human factor VIII variant.单次外周静脉给予编码新型人凝血因子 VIII 变体的 rAAV 载体后 FVIII 的治疗水平。
Blood. 2013 Apr 25;121(17):3335-44. doi: 10.1182/blood-2012-10-462200. Epub 2013 Feb 20.
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Adenovirus-associated virus vector-mediated gene transfer in hemophilia B.腺相关病毒载体介导的乙型血友病基因转移。
N Engl J Med. 2011 Dec 22;365(25):2357-65. doi: 10.1056/NEJMoa1108046. Epub 2011 Dec 10.
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Good manufacturing practice production of self-complementary serotype 8 adeno-associated viral vector for a hemophilia B clinical trial.用于血友病 B 临床试验的自互补血清型 8 腺相关病毒载体的良好生产规范生产。
Hum Gene Ther. 2011 May;22(5):595-604. doi: 10.1089/hum.2010.202.
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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.经系统给予编码人凝血因子 IX 的自我互补型 AAV 载体（假型 5 型和 8 型衣壳蛋白）后，其长期安全性和疗效。
Mol Ther. 2011 May;19(5):876-85. doi: 10.1038/mt.2010.274. Epub 2011 Jan 18.
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Codon optimization of human factor VIII cDNAs leads to high-level expression.人凝血因子 VIII cDNA 的密码子优化导致高水平表达。
Blood. 2011 Jan 20;117(3):798-807. doi: 10.1182/blood-2010-05-282707. Epub 2010 Nov 1.
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Analysis of factor VIII mediated suppression of lentiviral vector titres.VIII因子介导的慢病毒载体滴度抑制分析。
Gene Ther. 2008 Feb;15(4):289-97. doi: 10.1038/sj.gt.3303080. Epub 2007 Nov 29.
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