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Updated: Jan 2, 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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Gene Editing Expands the Donor Pool for CCR5-Negative Stem Cell Transplants
1Keck School of Medicine of the University of Southern California, Los Angeles, CA, USA.
Cell Stem Cell
|December 7, 2019
Summary
CRISPR gene editing can disrupt the CCR5 gene in stem cells, potentially expanding donor options for HIV therapy. This approach offers a proof of concept for enhancing resistance in cell therapies for HIV patients.
Area of Science:
- Gene editing technologies
- HIV/AIDS research
- Stem cell biology
Background:
- Cellular therapies for HIV are limited by donor availability.
- Naturally occurring CCR5 mutations confer HIV resistance.
- CCR5 is a co-receptor for HIV entry into cells.
Purpose of the Study:
- To investigate a CRISPR-based method for CCR5 disruption.
- To assess the feasibility of expanding donor pools for HIV cell therapy.
Main Methods:
- Utilized CRISPR gene editing technology.
- Targeted disruption of the CCR5 gene in hematopoietic stem cells.
- Pre-transplant modification of stem cells.
Main Results:
- Demonstrated a proof-of-concept for CRISPR-mediated CCR5 disruption.
- Showcased a potential method to enhance stem cell resistance to HIV.
Conclusions:
- CRISPR offers a viable strategy for modifying hematopoietic stem cells.
- This method could broaden the accessibility of cell-based HIV treatments.
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