Objective: In this review, the hematopoietic stem cell is defined and
the change of this definition over the past decades is elucidated. Bes
ides we discuss how the applications of stem cell transplantation have
changed in the same period of time. In addition to the biology of hem
atopoietic stem cells, the most recent applications in gene transfer a
nd gene therapy will be discussed. Data Sources: Review of the literat
ure and results of studies performed at the Fred Hutchinson Cancer Res
earch Center in Seattle, USA. Selection Criteria: Peer review journals
and papers relevant to the topic. Results: Based on the initial radia
tion experiments in mice, the hematopoietic stem cell was defined by i
ts biological properties and later by the expression of certain surfac
e antigens, as well as the absence of lineage-specific markers. Quite
recently, more and more evidence has emerged that hematopoietic stem c
ells can be isolated from peripheral blood and that there might be a c
ommon progenitor of hematopoiesis and the marrow microenvironment. Hem
atopoietic stem cells are also a useful aid for gene therapy in a broa
der range of immunodeficiencies and metabolic disorders. Conclusions:
Peripheral blood stem cells transplantation and gene therapy will be u
seful tools to treat a broader spectrum of diseases using uncommitted
very early progenitor cells isolated from peripheral blood.