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Updated: Apr 3, 2026
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CRISPR/Cas9 Gene Editing of Hematopoietic Stem and Progenitor Cells for Gene Therapy Applications
Published on: August 9, 2022
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Pluripotent stem cell based gene therapy for hematological diseases.
Stijn Vanhee1, Bart Vandekerckhove1
1Department of Clinical Chemistry, Microbiology and Immunology, Ghent University, Belgium.
Critical Reviews in Oncology/Hematology
|September 19, 2015
Summary
Gene therapy for inherited blood diseases can be improved by combining induced pluripotent stem cells (iPSC) and gene editing. This approach offers a potential alternative to current treatments, addressing safety and efficacy concerns.
Area of Science:
- Biotechnology
- Hematology
- Genetics
Background:
- Allogeneic stem cell transplantation is standard for severe inherited hematopoietic diseases.
- Current gene therapy uses viral vectors to insert functional genes into hematopoietic stem and progenitor cells (HSPC), but has limitations.
Purpose of the Study:
- To review recent literature on combining induced pluripotent stem cells (iPSC) and nuclease-mediated gene editing for treating inherited hematopoietic diseases.
- To discuss the clinical applicability of this combined technology and identify future research directions.
Main Methods:
- Review of recent scientific literature.
- Discussion of the combination of iPSC technology and gene editing for HSPC modification.
- Analysis of current clinical applications and future research needs.
Main Results:
- Induced pluripotent stem cells (iPSC) can be generated from patient samples.
- Nuclease-mediated gene editing allows for precise correction of genetic defects in HSPC.
- The combination of iPSC and gene editing shows promise for clinical application.
Conclusions:
- Combining iPSC technology with gene editing offers a novel strategy for treating inherited hematopoietic diseases.
- This approach may overcome safety and efficacy issues associated with current gene therapy.
- Further research is needed to fully realize the clinical potential of this combined technology.
Keywords:
Gene therapyHematological diseasesHematopoiesisIPSCPluripotent stem cellsStem cellsTailored nucleaseshESCMore Related Videos
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