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CRISPR/Cas9 Gene Editing of Hematopoietic Stem and Progenitor Cells for Gene Therapy Applications
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Current and future gene therapies for hemoglobinopathies.
Christian Brendel1,2,3, David A Williams1,2,3
1Division of Hematology/Oncology, Boston Children's Hospital.
Current Opinion in Hematology
|March 25, 2020
Summary
Novel gene therapies offer potential cures for sickle cell disease (SCD) and beta-thalassemia. While early trials show promise, long-term safety and efficacy of these genetic treatments require further investigation.
Area of Science:
- Hematology
- Genetics
- Molecular Biology
Background:
- Sickle cell disease (SCD) and beta-thalassemia are significant genetic blood disorders.
- Current treatments for SCD and beta-thalassemia are largely supportive and do not offer a cure.
Purpose of the Study:
- To summarize conventional therapies for SCD and beta-thalassemia.
- To review emerging genetic therapies for these beta-hemoglobinopathies.
Main Methods:
- Review of current literature on conventional and genetic therapies.
- Analysis of ongoing clinical trials for gene-based treatments.
Main Results:
- Gene therapy, including viral vector and gene editing approaches, is a developing treatment modality.
- Phase I clinical studies are underway for several novel therapies, including those inducing fetal hemoglobin.
Conclusions:
- Early clinical trials indicate short-term benefits for some gene therapies.
- Long-term safety and efficacy data are still needed.
- Future therapies may focus on precise DNA mutation correction.
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