Abstract

Because of their capacity for self-renewal and multilineage differentiation into all types of mature blood cells, hematopoietic stem cells (HSCs) are highly attractive, not only as targets of gene therapy but also as cell sources for regeneration medicine. Hence, efficient transduction into HSCs holds promise for treating a number of diseases that result from abnormal blood cell function, and it would provide a powerful approach for understanding their basic biology and for manipulating their properties in vitro and in vivo.

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