Gene therapy for hemophilia: the clot thickens.
作者信息
High Katherine A
机构信息
Spark Therapeutics, Philadelphia , PA 19104.
出版信息
Hum Gene Ther. 2014 Nov;25(11):915-22. doi: 10.1089/hum.2014.2541.
Abstract
摘要
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Hum Gene Ther. 2014 Nov;25(11):915-22. doi: 10.1089/hum.2014.2541.
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本文引用的文献
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Immune responses to AAV vectors: overcoming barriers to successful gene therapy.腺相关病毒载体的免疫反应：克服成功基因治疗的障碍。
Blood. 2013 Jul 4;122(1):23-36. doi: 10.1182/blood-2013-01-306647. Epub 2013 Apr 17.
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The gene therapy journey for hemophilia: are we there yet?基因治疗血友病之旅：我们到了吗？
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Factor IX expression in skeletal muscle of a severe hemophilia B patient 10 years after AAV-mediated gene transfer.接受 AAV 介导的基因转移 10 年后，严重血友病 B 患者骨骼肌中因子 IX 的表达。
Blood. 2012 Mar 29;119(13):3038-41. doi: 10.1182/blood-2011-09-382317. Epub 2012 Jan 23.
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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。
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Cell phones and landlines: the impact of gene therapy on the cost and availability of treatment for hemophilia.手机与固定电话：基因疗法对血友病治疗成本及可及性的影响
Mol Ther. 2011 Oct;19(10):1749-50. doi: 10.1038/mt.2011.203.
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Therapeutic in vivo gene transfer for genetic disease using AAV: progress and challenges.利用 AAV 进行体内基因治疗遗传性疾病：进展与挑战。
Nat Rev Genet. 2011 May;12(5):341-55. doi: 10.1038/nrg2988.
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X-linked thrombophilia with a mutant factor IX (factor IX Padua).伴有突变型凝血因子IX（帕多瓦凝血因子IX）的X连锁血栓形成倾向
N Engl J Med. 2009 Oct 22;361(17):1671-5. doi: 10.1056/NEJMoa0904377.
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Gene therapy for haemophilia.血友病的基因治疗。
Br J Haematol. 2008 Mar;140(5):479-87. doi: 10.1111/j.1365-2141.2007.06942.x.
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Adeno-associated virus type 2 (AAV2) capsid-specific cytotoxic T lymphocytes eliminate only vector-transduced cells coexpressing the AAV2 capsid in vivo.2型腺相关病毒（AAV2）衣壳特异性细胞毒性T淋巴细胞在体内仅消除共表达AAV2衣壳的载体转导细胞。
J Virol. 2007 Jul;81(14):7540-7. doi: 10.1128/JVI.00529-07. Epub 2007 May 2.
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