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Applications of hematopoietic stem cells and gene transfer
1Pathologisches Institut, Ludwig-Maximilians-Universität, München, Germany.
Insights
Hematopoietic stem cells (HSCs) have evolved in definition and application. Recent advances show HSCs in peripheral blood can be used for gene therapy, expanding treatment options for various disorders.
Area of Science:
- Hematology
- Stem Cell Biology
- Gene Therapy
Background:
- The definition of hematopoietic stem cells (HSCs) has evolved over decades.
- Stem cell transplantation applications have similarly transformed.
- Recent research explores HSCs in gene transfer and therapy.
Purpose of the Study:
- To define hematopoietic stem cells and trace the evolution of their definition.
- To elucidate changes in stem cell transplantation applications.
- To discuss recent advancements in HSC biology and gene therapy.
Main Methods:
- Literature review of peer-reviewed journals and relevant papers.
- Inclusion of results from studies conducted at Fred Hutchison Cancer Research Center.
Main Results:
- HSC definition shifted from biological properties to surface antigen expression and marker absence.
- Emerging evidence supports HSC isolation from peripheral blood and a common progenitor concept.
- HSCs are valuable for gene therapy in immunodeficiencies and metabolic disorders.
Conclusions:
- Peripheral blood stem cell transplantation and gene therapy offer expanded treatment potential.
- Uncommitted progenitor cells from peripheral blood are key to future therapies.
- These approaches will broaden the spectrum of treatable diseases.
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.