Related Experiment Video
Updated: May 4, 2026

CRISPR/Cas9 Gene Editing of Hematopoietic Stem and Progenitor Cells for Gene Therapy Applications
Published on: August 9, 2022
Gene-modified hematopoietic stem cells for cancer immunotherapy
Sarah Larson1, Satiro N De Oliveira2
1Department of Medicine; Division of Hematology/Oncology; David Geffen School of Medicine at the University of California, Los Angeles; Los Angeles, CA USA.
Cancer immunotherapy shows promise with gene therapy introducing chimeric antigen receptors (CARs) and T cell receptors (TCRs). Modifying hematopoietic stem cells (HSCs) with these receptors and a suicide gene offers enhanced safety and control for improved patient outcomes.
Area of Science:
- Oncology
- Immunology
- Gene Therapy
Background:
- Cancer immunotherapy has rapidly expanded, yielding positive early results.
- Chimeric antigen receptors (CARs) and T cell receptors (TCRs) introduced via gene therapy offer novel treatment avenues.
- Current CAR and TCR T cell therapies face limitations that necessitate further innovation.
Purpose of the Study:
- To explore the modification of hematopoietic stem cells (HSCs) for CAR and TCR expression.
- To investigate the potential of HSC modification to overcome limitations of current T cell-based immunotherapies.
- To assess the role of suicide gene technology in enhancing the safety and control of HSC modification.
Main Methods:
- Utilizing modern gene therapy techniques for HSC modification.
- Introducing chimeric antigen receptors (CARs) and T cell receptors (TCRs) into HSCs.
- Incorporating a suicide gene into the modified HSCs for safety control.
Main Results:
- Hematopoietic stem cells (HSCs) can be modified to express CARs and TCRs.
- The incorporation of a suicide gene enhances safety and control over HSC modification.
- This approach presents a promising strategy to overcome existing immunotherapy limitations.
Conclusions:
- Modification of hematopoietic stem cells (HSCs) offers a potential advancement in cancer immunotherapy.
- The use of suicide genes alongside CAR/TCR expression in HSCs improves therapeutic safety and precision.
- This strategy holds promise for developing more effective and controlled cancer immunotherapies.
Related Concept Videos
Stem Cell Therapy for Tissue Regeneration
Types of Stem Cells used in Stem Cell Therapy
The two main cell...
Regulation of Hematopoietic Stem Cells
Tumor Immunotherapy
Mesenchymal Stem Cells
Bone Marrow Sampling and Transplants
The transplant begins with high doses of chemotherapy and radiation treatment, which aim to destroy...
Targeted Cancer Therapies
There are several types of targeted therapies against...

