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Updated: Oct 21, 2025
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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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Recent Progress in Genome Editing for Gene Therapy Applications: The French Perspective
Mario Amendola1,2, Aurélie Bedel3,4,5, Ana Buj-Bello1,2
1Genethon, Evry, France.
Human Gene Therapy
|September 8, 2021
Summary
Genome editing technologies, like CRISPR/Cas9, offer future cures for genetic diseases. French researchers are advancing therapeutic genome editing for blood disorders, muscular dystrophy, and cancer, while addressing safety and delivery challenges.
Area of Science:
- Biotechnology
- Genetics
- Molecular Biology
Background:
- CRISPR/Cas9 and related genome editing tools have revolutionized basic science and gene therapy.
- These tools enable precise genomic modifications for treating genetic diseases.
- Therapeutic genome editing holds promise as a future medicine.
Purpose of the Study:
- To review recent progress in therapeutic genome editing by French researchers.
- To place these advances within the broader context of global developments.
- To highlight applications in blood diseases, muscular dystrophy, and cancer immunotherapy.
Main Methods:
- Focus on hematopoietic stem cell gene editing for blood and lysosomal storage diseases.
- Review genome editing therapies for muscular dystrophy.
- Discuss T cell gene editing for enhancing chimeric antigen receptor T cell therapy against cancer.
Main Results:
- Exploration of gene editing strategies for red blood cell disorders, coagulation defects, and lysosomal storage diseases.
- Assessment of genome editing for muscular dystrophy treatment.
- Evaluation of T cell gene editing's potential in cancer immunotherapy.
Conclusions:
- Genome editing shows significant potential for treating various genetic disorders and cancers.
- Technical challenges, including off-target effects and delivery methods, require further solutions.
- Novel systems like nickases, base editors, and prime editors are improving precision and safety.
- Addressing cellular toxicity from delivery systems is crucial for clinical implementation.
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