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Updated: Mar 27, 2026
08:32
CRISPR/Cas9 Gene Editing of Hematopoietic Stem and Progenitor Cells for Gene Therapy Applications
Published on: August 9, 2022
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Genome-editing Technologies for Gene and Cell Therapy.
Morgan L Maeder1, Charles A Gersbach2,3,4
1Editas Medicine, Cambridge, Massachusetts, USA.
Summary
Genome editing precisely manipulates DNA for therapeutic effects, correcting mutations and adding genes. This review covers genome editing technologies and their applications in gene and cell therapy, highlighting future prospects.
Area of Science:
- Biotechnology
- Genetics
- Molecular Biology
Background:
- Traditional gene therapy involved adding genes.
- Genome editing offers precise DNA manipulation for therapeutic benefit.
- Technologies allow correction, addition, or removal of genomic sequences.
Purpose of the Study:
- To review genome editing mechanisms and platforms.
- To summarize applications in gene and cell therapy.
- To discuss challenges and future directions.
Main Methods:
- Review of nuclease-based genome editing platforms.
- Description of zinc finger nucleases (ZFNs).
- Description of transcription activator-like effector nucleases (TALENs).
- Description of meganucleases.
- Description of CRISPR/Cas9 system.
Main Results:
- Genome editing enables precise correction of disease-causing mutations.
- Therapeutic genes can be inserted at specific genomic locations.
- Deleterious genes or sequences can be removed.
- Applications include antiviral strategies, immunotherapies, and treating monogenic disorders.
Conclusions:
- Genome editing represents a transformative approach to gene and cell therapy.
- Significant progress has been made in applying these technologies.
- Challenges remain, but future prospects are promising.
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