News Column

Findings from French National Institute of Health and Medical Research (INSERM) Update Understanding of Stem Cells

June 5, 2014

By a News Reporter-Staff News Editor at Gene Therapy Weekly -- Investigators publish new report on Stem Cell Research. According to news reporting originating in Paris, France, by NewsRx journalists, research stated, "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)."

The news reporters obtained a quote from the research from the French National Institute of Health and Medical Research (INSERM), "To develop this approach, we chose an Artemis knockout (Art(-/-)) mouse in which exon 12 of the Artemis gene had been replaced by an I-Sce1 recognition site. The I-Sce1 enzyme and the Artemis correction template were each delivered by a self-inactivating (SIN)-integrase-defective lentiviral vector (SIN-IDLV-CMV-ISce1 and SIN-IDLV-Art, respectively). Transduction of Art-/- mHSCs with the two vectors successfully reverted the Art(-/-) phenotype in 2 of our 10 experiments. Even though the potential for genotoxicity has yet to be evaluated, this new approach to gene editing appears to be promising."

According to the news reporters, the research concluded: "Improving the efficacy of this approach will require further technical work."

For more information on this research see: Variable correction of Artemis deficiency by I-Sce1-meganuclease-assisted homologous recombination in murine hematopoietic stem cells. Gene Therapy, 2014;21(5):529-532. Gene Therapy can be contacted at: Nature Publishing Group, Macmillan Building, 4 Crinan St, London N1 9XW, England. (Nature Publishing Group -; Gene Therapy -

Our news correspondents report that additional information may be obtained by contacting J. Riviere, INSERM, APHP, CIC Biotherapy GHU West, Paris, France. Additional authors for this research include J. Hauer, L. Poirot, J. Brochet, P. Souque, K. Mollier, A. Gouble, P. Charneau, A. Fischer, F. Paques, J.P. de Villartay and M. Cavazzana (see also Stem Cell Research).

Keywords for this news article include: Biotechnology, Paris, France, Europe, Hematology, Gene Therapy, Bioengineering, Bone Marrow Cells, Stem Cell Research, Hematopoietic Stem Cells

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Source: Gene Therapy Weekly

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