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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 for immune cell therapies.
Stefanie R Bailey1,2, Marcela V Maus3,4
1Cellular Immunotherapy Program, Cancer Center, Massachusetts General Hospital, Boston, MA, USA.
Nature Biotechnology
|June 5, 2019
Summary
Chimeric antigen receptor (CAR) T cell therapy shows promise for B cell malignancies. Advances in immune cell engineering offer new solutions for cancer treatment, expanding to other cell types and diseases.
Area of Science:
- Immunology
- Genetics
- Oncology
Background:
- Chimeric antigen receptor (CAR) T cell therapies targeting CD19 have achieved significant clinical success in B cell malignancies.
- The field of immune cell engineering is rapidly advancing to address challenges like antigen selection, tumor microenvironment, and therapy logistics.
Purpose of the Study:
- To review innovations in immune cell engineering for cancer therapy.
- To discuss the application of these technologies to various immune cells and diseases.
- To highlight preclinical and early clinical findings of next-generation cell therapies.
Main Methods:
- Review of current literature and clinical trial data on engineered cell therapies.
- Discussion of advancements in CAR design, gene transfer, and gene-editing technologies.
- Exploration of applications beyond T cells, including NK cells, hematopoietic stem cells, and iPSCs.
Main Results:
- Engineered T cells demonstrate sophisticated sensing circuits and synthetic functionalities.
- Development of off-the-shelf, universal cellular products is progressing.
- Next-generation approaches are showing promising early translational results in clinical trials.
Conclusions:
- Engineered cell therapies hold significant potential to transform the treatment of various cancers and other diseases.
- Continued innovation in gene-modified and gene-edited cell therapies is expected.
- The application of these technologies to diverse cell types broadens therapeutic possibilities.
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