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Advances in the Application of CRISPR-Cas9 in Stem Cell Therapy
- 1 China Pharmaceutical University, Nanjing, China
* Author to whom correspondence should be addressed.
Abstract
Stem cell research has advanced rapidly, offering promising treatments for refractory diseases due to their unique capabilities for self-renewal and pluripotent differentiation. Stem cells play pivotal roles in treating genetic disorders, neurodegenerative diseases (NDDs), cardiovascular conditions, and cancer. In genetic diseases, combining stem cells with gene-editing tools like CRISPR-Cas9 enables precise correction of pathogenic genes, while healthy stem cells repair tissue by replacing diseased cells. For NDDs, iPSCs can differentiate into dopaminergic neurons to replace damaged brain cells and enhance neural regeneration. In cardiovascular diseases, they promote myocardial and vascular repair. In cancer, stem cells boost anti-tumor immunity and deliver drugs directly to tumor sites, improving treatment efficacy. Despite these breakthroughs, challenges persist. High-quality stem cell production is limited, and controlling differentiation to prevent tumorigenesis remains critical. Allogeneic transplants risk immune rejection, and using embryonic stem cells raises ethical concerns. Regulatory frameworks and clinical standards are needed to ensure safety and efficacy, alongside addressing ethical and patient rights issues. With continued innovation, stem cell therapy is going to revolutionize medicine, offering novel methods for complex diseases and improving global health
Keywords
CRISPR-Cas9, cancer, stem cell, neurodegenerative disease
[1]. Takahashi, K., & Yamanaka, S. (2006). Induction of pluripotent stem cells from mouse embryonic and adult fibroblast cultures by defined factors. Cell, 126, 663-676.
[2]. Xu, R. L., et al. (2019). Correction of a pathogenic gene mutation in human embryos using CRISPR-Cas9.
[3]. Hochedlinger, K., & Plath, K. (2009). Epigenetic reprogramming and induced pluripotency. Development, 136, 509-523.
[4]. Wang, S. W., Gao, C., Zheng, Y. M., et al. (2022). Current applications and future perspective of CRISPR/Cas9 gene editing in cancer. Molecular Cancer, 21, 57.
[5]. Xiong, Z., & Jiang, Y. (2024). Research progress on methods to improve the efficacy of mesenchymal stem cell transplantation for treating liver cirrhosis. Journal of Gastroenterology and Hepatology, 33(10), 1401-1404.
[6]. Normile, D. (2017). China sprints ahead in CRISPR therapy race. Science, 358, 20-21.
[7]. Rupp, L. J., Schumann, K., Roybal, K. T., et al. (2017). CRISPR/Cas9-mediated PD-1 disruption enhances anti-tumor efficacy of human chimeric antigen receptor T cells. Scientific Reports, 7(1), 1-10.
[8]. Ou, X., Ma, Q., Yin, W., et al. (2021). CRISPR/Cas9 gene-editing in cancer immunotherapy: Promoting the present revolution in cancer therapy and exploring more. Frontiers in Cell and Developmental Biology, 9, 674467.
[9]. Feng, Y., Sassi, S., Shen, J. K., et al. (2015). Targeting Cdk11 in osteosarcoma cells using the CRISPR-Cas9 system. Journal of Orthopaedic Research, 33(2), 199-207.
[10]. Rath, J. A., & Arber, C. (2020). Engineering strategies to enhance TCR-based adoptive T cell therapy. Cells, 9(6), 1485.
Cite this article
Wu,H. (2025).Advances in the Application of CRISPR-Cas9 in Stem Cell Therapy.Theoretical and Natural Science,90,76-80.
Data availability
The datasets used and/or analyzed during the current study will be available from the authors upon reasonable request.
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Volume title: Proceedings of ICMMGH 2025 Workshop: Computational Modelling in Biology and Medicine
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