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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.

Abstract
摘要

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本文引用的文献

1
Hemophilia gene therapy: a Holy Grail found.
Mol Ther. 2011 Mar;19(3):427-8. doi: 10.1038/mt.2011.13.
3
The pleiotropic effects of natural AAV infections on liver-directed gene transfer in macaques.
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.
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.
Blood. 2009 Sep 3;114(10):2077-86. doi: 10.1182/blood-2008-07-167510. Epub 2009 Jun 8.
6
Gene therapy for haemophilia.
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.
Science. 2007 Jul 27;317(5837):477. doi: 10.1126/science.1142658.

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