Gene therapy, stress haematopoiesis and the risk of malignancy

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The immense potential of gene therapy has been confirmed by the successful treatment of immune deficiencies disorders. A key issue that has emerged however is the need to avoid potentially cancer-causing genetic damage to cells undergoing repair. Some of this risk can be linked to the gene repair technology used, but we have identified and propose to investigate another potentially important mechanism involving the unusually high levels of replication imposed on cells during disease correction.