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Induced pluripotent stem cells in clinical hematology: potentials, progress, and remaining obstacles
Athanasia D Panopoulos1, Juan C I Belmonte
1Gene Expression Laboratory, The Salk Institute for Biological Studies, La Jolla, California, USA.
Current Opinion in Hematology
|May 5, 2012
Summary
Induced pluripotent stem cell (iPSC) technology offers potential for patient-specific therapies in hematology. Advances in stem cell research are paving the way for clinical applications, though challenges remain in generating functional hematopoietic stem cells (HSCs).
Area of Science:
- Stem Cell Biology
- Hematology
- Reproductive Biology
Background:
- Induced pluripotent stem cell (iPSC) technology enables the generation of patient-specific cells for therapeutic applications.
- The field of hematology seeks to leverage iPSC technology for novel clinical treatments.
- Significant advancements in stem cell research are crucial for translating these possibilities into clinical practice.
Purpose of the Study:
- To review recent key developments in stem cell and hematopoietic research.
- To identify remaining limitations in translating induced pluripotent stem cell (iPSC) technologies into the hematology clinic.
- To discuss the potential of iPSC-based therapies for hematological disorders.
Main Methods:
- Review of recent scientific literature on stem cell reprogramming and hematopoiesis.
- Analysis of progress in identifying and expanding hematopoietic stem cells (HSCs).
- Evaluation of gene-targeting strategies for correcting disease-specific iPSCs.
Main Results:
- Identification of single-cell level hematopoietic stem cells (HSCs) with long-term engraftment potential.
- Improvements in ex-vivo expansion techniques for HSCs.
- Successful transdifferentiation of somatic cells into hematopoietic progenitors.
- Correction of genetic defects in disease-specific iPSCs using gene-targeting strategies.
Conclusions:
- Overcoming challenges in generating fully functional HSCs in vitro is anticipated through pluripotent stem cell differentiation, ex-vivo expansion, or transdifferentiation.
- Understanding genetic/epigenetic variations in iPSCs is critical for gene-targeted therapies.
- Progress in reprogramming and hematopoietic fields provides a foundation for treating hematological disorders with iPSC technology.
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