Efficient genome editing in hematopoietic stem cells with helper-dependent Ad5/35 vectors expressing site-specific endonucleases under microRNA regulation

Genome editing with site-specific endonucleases has implications for basic biomedical research as well as for gene therapy. We generated helper-dependent, capsid-modified adenovirus (HD-Ad5/35) vectors for zinc-finger nuclease (ZFN)â or transcription activator-like effector nuclease (TALEN)âmediated...

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Main Authors: Kamola Saydaminova, Xun Ye, Hongjie Wang, Maximilian Richter, Martin Ho, HongZhuan Chen, Ning Xu, Jin-Soo Kim, Eirini Papapetrou, Michael C Holmes, Philip D Gregory, Donna Palmer, Philip Ng, Anja Ehrhardt, André Lieber
Format: Article
Language:English
Published: Elsevier 2015-01-01
Series:Molecular Therapy: Methods & Clinical Development
Online Access:http://www.sciencedirect.com/science/article/pii/S232905011630002X