Abstract

Hematopoietic stem cells maintain life long blood generation during homeostasis and stress. Factors controlling the key characteristics of these cells such as self-renewal, quiescence and the potential to form various blood lineages are poorly understood. We reasoned that genes found to be located near common sites of vector insertion in clinical gene therapy studies might have important functions in this regard. The DNA binding protein, high mobility Group AT hook 2 (HMGA2) is one such factor. HMGA2 activation coupled with a myeloid biased clonal dominance of stem/progenitor cells was observed in a clinical gene therapy trial for β-thalassemia.

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