Site-specific integration and tailoring of cassette design for sustainable gene transfer

被引:0
作者
Angelo Lombardo
Daniela Cesana
Pietro Genovese
Bruno Di Stefano
Elena Provasi
Daniele F Colombo
Margherita Neri
Zulma Magnani
Alessio Cantore
Pietro Lo Riso
Martina Damo
Oscar M Pello
Michael C Holmes
Philip D Gregory
Angela Gritti
Vania Broccoli
Chiara Bonini
Luigi Naldini
机构
[1] San Raffaele Telethon Institute for Gene Therapy,Division of Regenerative Medicine
[2] Gene Therapy and Stem Cells,Division of Neurosciences
[3] San Raffaele Institute,Division of Regenerative Medicine
[4] Vita-Salute San Raffaele University,undefined
[5] Stem Cell and Neurogenesis Unit,undefined
[6] San Raffaele Institute,undefined
[7] Experimental Hematology Unit,undefined
[8] Gene Therapy and Stem Cells,undefined
[9] Program in Immunology and Bio-immunotherapy of Cancer,undefined
[10] San Raffaele Scientific Institute,undefined
[11] Sangamo BioSciences Inc.,undefined
[12] Present address: Hematopoietic Stem Cell Biology and Differentiation Group,undefined
[13] Department of Differentiation and Cancer Centre for Genomic Regulation,undefined
[14] Barcelona,undefined
[15] Spain.,undefined
来源
Nature Methods | 2011年 / 8卷
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学科分类号
摘要
Presented is an experimental analysis of the stability of transgene expression, the perturbation of endogenous expression and the perturbation of epigenetic organization upon site-directed delivery of transgenes to the CCR5 and AAVS1 loci in human cells. It provides guidelines for optimal cassette design for stable and nonperturbative gene transfer.
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页码:861 / 869
页数:8
相关论文
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