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Gene therapy activates EVI1, destabilizes chromosomes

Nature Medicine volume 16, pages 163–165 (2010)Cite this article

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One hazard of gene therapy is that the vector will insert into an inappropriate location, causing aberrant expression of genes that can lead to disease. A new study reveals how such events occurred in a recent gene therapy trial using a vector that has now fallen out of favor (pages 198–204).

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Figure 1: Progression of gene correction and clinical outcome in CGD clinical trial.

Katie Vicari

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Authors and Affiliations

  1. Cynthia E. Dunbar and Andre Larochelle are in the Hematology Branch, National Heart, Lung, and Blood Institute, US National Institutes of Health, Bethesda, Maryland, USA.,

    Cynthia E Dunbar & Andre Larochelle

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  1. Cynthia E Dunbar
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  2. Andre Larochelle
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Correspondence to Andre Larochelle.

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The authors declare no competing financial interests.

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Dunbar, C., Larochelle, A. Gene therapy activates EVI1, destabilizes chromosomes. Nat Med 16, 163–165 (2010). https://doi.org/10.1038/nm0210-163

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