Article
Promoterless gene targeting without nucleases ameliorates haemophilia B in mice.
Nature - 15 Jan 2015
Barzel A, Paulk N K, Shi Y, Huang Y, Chu K, Zhang F, Valdmanis P N, Spector L P, Porteus M H, Gaensler K M, Kay M A
Abstract excerpt
Site-specific gene addition can allow stable transgene expression for gene therapy. When possible, this is preferred over the use of promiscuously integrating vectors, which are sometimes associated with clonal expansion and oncogenesis. Site-specific endonucleases that can induce high rates of targeted genome editing are finding increasing applications in biological discovery and gene therapy. However, two...
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