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Gene transfer into hematopoietic stem cells

The ability to reliably transfer genes into hematopoietic stem cells with long‐term repopulating potential and to selectively express such genes would allow genetic therapy for diseases such as sickle cell anemia and immunologic deficiencies due to T‐cell defects, including acquired immune deficiency syndrome (AIDS). Understanding the biology of the hematopoietic stem cell is a key element in realizing the full therapeutic potential of gene insertion strategies. Current techniques have efficiency rates of gene insertion of approximately 10% to 20% into murine stem cells and 1% to 5% into primate stem cells. Many challenges, some biologic and some logistic, remain before gene transfer protocols that are successful in the mouse model can be extended to humans.