Applications of hematopoietic stem cells and gene transfer.

Abstract

Objective: In this review, the hematopoietic stem cell is defined and the change of this definition over the past decades is elucidated. Besides we discuss how the applications of stem cell transplantation have changed in the same period of time. In addition to the biology of hematopoietic stem cells, the most recent applications in gene transfer and gene therapy will be discussed.

Data sources: Review of the literature and results of studies performed at the Fred Hutchison Cancer Research Center in Seattle, USA.

Selection criteria: Peer review journals and papers relevant to the topic.

Results: Based on the initial radiation experiments in mice, the hematopoietic stem cell was defined by its biological properties and later by the expression of certain surface antigens, as well as the absence of lineage-specific markers. Quite recently, more and more evidence has emerged that hematopoietic stem cells can be isolated from peripheral blood and that there might be a common progenitor of hematopoiesis and the marrow microenvironment. Hematopoietic stem cells are also a useful aid for gene therapy in a broader range of immunodeficiencies and metabolic disorders.

Conclusions: Peripheral blood stem cells transplantation and gene therapy will be useful tools to treat a broader spectrum of diseases using uncommitted very early progenitor cells isolated from peripheral blood.