[Technology for gene transfer into hematopoietic stem cells (Part 2)].

A hematopoietic stem cell is considered to be one of the ideal targets for gene therapy, and there is expectation that gene therapy will be established based on the technology of hematopoietic stem cell transplantation. However, in recent clinical trials of stem cell gene therapy for monogenic diseases, significant clinical improvement has not been reported. One of the main obstacles is the low efficiency of gene transfer into hematopoietic stem cells. Many investigators have been trying to improve the transduction efficiency to the clinically applicable level. Another approach to solve this problem is to develop the method for selective expansion of transduced hematopoietic stem cells in vivo. We are currently developing novel regulatory genes (selective amplifier genes) for stem cell gene therapy.