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Updated: Nov 21, 2025
CRISPR/Cas9 Gene Editing of Hematopoietic Stem and Progenitor Cells for Gene Therapy Applications
Published on: August 9, 2022
CRISPR/Cas9 gene editing for curing sickle cell disease
1Department of Bioengineering, Rice University, 6500 Main St, Houston, TX, 77030, USA.
Insights
Gene editing offers a potential cure for sickle cell disease (SCD) by correcting the HBB gene mutation or inducing fetal hemoglobin. This approach could provide a permanent solution for all SCD patients.
Area of Science:
- Hematology
- Genetics
- Molecular Biology
Background:
- Sickle cell disease (SCD) is a prevalent monogenic blood disorder causing severe pain, organ damage, and reduced lifespan.
- Current treatments for SCD are limited, with hematopoietic stem cell transplantation being the only cure, often requiring a matched donor.
Purpose of the Study:
- To review the application of CRISPR/Cas9 gene-editing technology for curing sickle cell disease.
- To explore two primary gene-editing strategies: direct correction of the HBB mutation and induction of fetal hemoglobin.
Main Methods:
- Focuses on CRISPR/Cas9 gene-editing techniques applied to hematopoietic stem and progenitor cells.
- Discusses ex vivo genetic modification of autologous cells for subsequent transplantation.
Main Results:
- CRISPR/Cas9 shows promise in correcting the SCD-causing mutation in the beta-globin (HBB) gene.
- Gene editing can induce fetal hemoglobin production, effectively reversing red blood cell sickling.
Conclusions:
- CRISPR/Cas9 gene editing presents a potential curative therapy for SCD, overcoming donor limitations and graft-versus-host disease.
- Further research and development are crucial to address challenges and realize the full potential of gene-editing therapies for SCD.
Abstract:
Sickle cell disease (SCD) is the most common monogenic blood disorder marked by severe pain, end-organ damage, and early mortality. Treatment options for SCD remain very limited. There are only four FDA approved drugs to reduce acute complications. The only curative therapy for SCD is hematopoietic stem cell transplantation, typically from a matched, related donor. Ex vivo engineering of autologous hematopoietic stem and progenitor cells followed by transplantation of genetically modified cells potentially provides a permanent cure applicable to all patients regardless of the availability of suitable donors and graft-vs-host disease. In this review, we focus on the use of CRISPR/Cas9 gene-editing for curing SCD, including the curative correction of SCD mutation in β-globin (HBB) and the induction of fetal hemoglobin to reverse sickling. We summarize the major achievements and challenges, aiming to provide a clearer perspective on the potential of gene-editing based approaches in curing SCD.
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