Genotoxicity in Mice Following AAV Gene Delivery: A Safety Concern for Human Gene Therapy?
作者信息
Chandler Randy J, LaFave Matthew C, Varshney Gaurav K, Burgess Shawn M, Venditti Charles P
机构信息
Molecular Genomics and Metabolic Genetics Branch, National Human Genome Research Institute, National Institutes of Health, Bethesda, Maryland, USA.
Translational and Functional Genomics Branch, National Human Genome Research Institute, National Institutes of Health, Bethesda, Maryland, USA.
出版信息
Mol Ther. 2016 Feb;24(2):198-201. doi: 10.1038/mt.2016.17.
Abstract
摘要
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2
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Mol Ther. 2015 Nov;23(11):1673-1675. doi: 10.1038/mt.2015.182.
3
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Nat Genet. 2015 Oct;47(10):1104-5. doi: 10.1038/ng.3407.
4
Recurrent AAV2-related insertional mutagenesis in human hepatocellular carcinomas.
Nat Genet. 2015 Oct;47(10):1187-93. doi: 10.1038/ng.3389. Epub 2015 Aug 24.
5
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J Clin Invest. 2015 Feb;125(2):870-80. doi: 10.1172/JCI79213. Epub 2015 Jan 20.
6
Long-term correction of Sandhoff disease following intravenous delivery of rAAV9 to mouse neonates.
Mol Ther. 2015 Mar;23(3):414-22. doi: 10.1038/mt.2014.240. Epub 2014 Dec 17.
7
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Bioinformatics. 2014 Apr 1;30(7):1003-5. doi: 10.1093/bioinformatics/btt637. Epub 2013 Nov 13.
8
Recombinant adeno-associated virus integration sites in murine liver after ornithine transcarbamylase gene correction.
Hum Gene Ther. 2013 May;24(5):520-5. doi: 10.1089/hum.2012.112.
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Identification of rtl1, a retrotransposon-derived imprinted gene, as a novel driver of hepatocarcinogenesis.
PLoS Genet. 2013 Apr;9(4):e1003441. doi: 10.1371/journal.pgen.1003441. Epub 2013 Apr 4.
10
No tumour-initiating risk associated with scAAV transduction in newborn rat liver.
Gene Ther. 2013 Jul;20(7):779-84. doi: 10.1038/gt.2013.7. Epub 2013 Jan 31.
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