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A barrel of monkeys: scAAV8 gene therapy for hemophilia in nonhuman primates.

作者信息

Chandler Randy J, Venditti Charles P

机构信息

Organic Acid Research Section, Genetics and Molecular Biology Branch, National Human Genome Research Institute, National Institutes of Health, Bethesda, Maryland 20893, USA.

出版信息

Mol Ther. 2011 May;19(5):826-7. doi: 10.1038/mt.2011.73.

DOI:10.1038/mt.2011.73
PMID:21532610
原文链接:https://pmc.ncbi.nlm.nih.gov/articles/PMC3098648/
Abstract
摘要

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A barrel of monkeys: scAAV8 gene therapy for hemophilia in nonhuman primates.一群猴子:非人类灵长类动物中用于血友病的scAAV8基因疗法。
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Employing a gain-of-function factor IX variant R338L to advance the efficacy and safety of hemophilia B human gene therapy: preclinical evaluation supporting an ongoing adeno-associated virus clinical trial.利用功能获得性因子IX变体R338L提高B型血友病人类基因治疗的疗效和安全性:支持正在进行的腺相关病毒临床试验的临床前评估
Hum Gene Ther. 2015 Feb;26(2):69-81. doi: 10.1089/hum.2014.106. Epub 2015 Jan 21.
3
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。
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Complete correction of hemophilia B phenotype by FIX-Padua skeletal muscle gene therapy in an inhibitor-prone dog model.FIX-Padua 骨骼肌基因治疗纠正血友病 B 表型,抑制物倾向犬模型。
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Gene therapy for hemophilia B mice with scAAV8-LP1-hFIX.用scAAV8-LP1-hFIX对血友病B小鼠进行基因治疗。
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Sustained correction of disease in naive and AAV2-pretreated hemophilia B dogs: AAV2/8-mediated, liver-directed gene therapy.初治和经腺相关病毒2(AAV2)预处理的B型血友病犬疾病的持续纠正:AAV2/8介导的肝脏定向基因治疗。
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Recombinant adeno-associated virus delivers human factor IX in mice.重组腺相关病毒在小鼠体内递送人凝血因子IX。
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J Rare Dis Res Treat. 2016;1(2):8-10.
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Minimizing the inhibitory effect of neutralizing antibody for efficient gene expression in the liver with adeno-associated virus 8 vectors.最小化中和抗体对腺相关病毒 8 载体在肝脏中高效基因表达的抑制作用。
Mol Ther. 2013 Feb;21(2):318-23. doi: 10.1038/mt.2012.258. Epub 2012 Dec 18.

本文引用的文献

1
Hemophilia gene therapy: a Holy Grail found.血友病基因疗法:找到了圣杯。
Mol Ther. 2011 Mar;19(3):427-8. doi: 10.1038/mt.2011.13.
2
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.
3
The pleiotropic effects of natural AAV infections on liver-directed gene transfer in macaques.天然 AAV 感染对食蟹猴肝脏靶向基因转移的多效性影响。
Mol Ther. 2010 Jan;18(1):126-34. doi: 10.1038/mt.2009.245. Epub 2009 Nov 3.
4
Sustained transgene expression despite T lymphocyte responses in a clinical trial of rAAV1-AAT gene therapy.在一项rAAV1 - AAT基因治疗的临床试验中,尽管存在T淋巴细胞反应,但转基因仍持续表达。
Proc Natl Acad Sci U S A. 2009 Sep 22;106(38):16363-8. doi: 10.1073/pnas.0904514106. Epub 2009 Aug 12.
5
AAV-1-mediated gene transfer to skeletal muscle in humans results in dose-dependent activation of capsid-specific T cells.腺相关病毒1型介导的基因向人体骨骼肌的转移导致衣壳特异性T细胞的剂量依赖性激活。
Blood. 2009 Sep 3;114(10):2077-86. doi: 10.1182/blood-2008-07-167510. Epub 2009 Jun 8.
6
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.
7
AAV vector integration sites in mouse hepatocellular carcinoma.小鼠肝细胞癌中的腺相关病毒载体整合位点
Science. 2007 Jul 27;317(5837):477. doi: 10.1126/science.1142658.
8
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.经外周静脉输注自我互补腺相关病毒载体后,肝脏实现安全有效的转导,可在非人灵长类动物中稳定表达人FIX进行治疗。
Blood. 2007 Feb 15;109(4):1414-21. doi: 10.1182/blood-2006-03-010181. Epub 2006 Nov 7.
9
Successful transduction of liver in hemophilia by AAV-Factor IX and limitations imposed by the host immune response.腺相关病毒介导的因子IX对血友病患者肝脏的成功转导及宿主免疫反应带来的限制
Nat Med. 2006 Mar;12(3):342-7. doi: 10.1038/nm1358. Epub 2006 Feb 12.
10
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.包含新型肝脏特异性人凝血因子IX表达盒的自互补腺相关病毒载体能够高效转导小鼠和非人类灵长类动物的肝脏。
Blood. 2006 Apr 1;107(7):2653-61. doi: 10.1182/blood-2005-10-4035. Epub 2005 Dec 1.