Article
Variable correction of Artemis deficiency by I-Sce1-meganuclease-assisted homologous recombination in murine hematopoietic stem cells.
Gene therapy - 1 May 2014
Rivière J, Hauer J, Poirot L, Brochet J, Souque P, Mollier K, Gouble A, Charneau P, Fischer A, Pâques F, de Villartay J-P, Cavazzana M
Abstract excerpt
The correction of genetic mutations by homologous recombination is an attractive approach to gene therapy. We used the DNA double-strand breaks introduced by the site-specific endonuclease I-Sce1 as a means of increasing homologous recombination of an exogenous DNA template in murine hematopoietic stem cells (mHSCs). To develop this approach, we chose an Artemis knockout (Art(-/-)) mouse in which exon 12 of the...
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