The MDS1–EVI1 Gene Complex as a Retrovirus Integration Site: Impact on Behavior of Hematopoietic Cells and Implications for Gene Therapy

Abstract excerpt

Gene therapy trials have been performed with virus-based vectors that have the ability to integrate permanently into genomic DNA and thus allow prolonged expression of corrective genes after transduction of hematopoietic stem and progenitor cells. Adverse events observed during the X-linked severe combined immunodeficiency gene therapy trial revealed a significant risk of genotoxicity related to retrovirus vector...

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