Progress of gene-mediated precision immunotherapy in the treatment of acute myeloid leukemia

ZHAO Pengbo; ZHU Ying; YIN Lifang; XIN Xiaofei

doi:10.11665/j.issn.1000-5048.2023112703

Journal of China Pharmaceutical University > 2024 > 55(1): 53-62. > DOI: 10.11665/j.issn.1000-5048.2023112703

ZHAO Pengbo, ZHU Ying, YIN Lifang, et al. Progress of gene-mediated precision immunotherapy in the treatment of acute myeloid leukemia[J]. J China Pharm Univ, 2024, 55(1): 53 − 62. DOI: 10.11665/j.issn.1000-5048.2023112703

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Progress of gene-mediated precision immunotherapy in the treatment of acute myeloid leukemia

ZHAO Pengbo^{1, 2},
ZHU Ying^{1, 2},
YIN Lifang^{1, 2, ,},
XIN Xiaofei^{1, 2, ,}

1.
Department of Pharmaceutics, School of Pharmacy, China Pharmaceutical University, Nanjing 210009
2.
Jiangsu Provincial Engineering Research Center for R & D and Evaluation of Intelligent Drugs and Key Functional Excipients, Nanjing 210009, China

Funds: This study was supported by the National Natural Science Foundation of China (No.82304410); the Natural Science Foundation of Jiangsu Province (No.BK20221045); the National Center of Technology Innovation for Biopharmaceuticals (No.NCTIB2022HS01015); and “Double First-Class” Initiative Program in China Pharmaceutical University

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Received Date: November 26, 2023
Available Online: March 05, 2024

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Abstract

Abstract

Acute myeloid leukemia (AML) is a disease caused by abnormal cloning of hematopoietic stem cells in the bone marrow, which leads to accumulation of a large number of abnormally differentiated myeloid cells. It is difficult to cure by traditional treatment. The successful application of chimeric antigen receptor T cell (CAR-T) immunotherapy indicates that the treatment of hematological tumors has entered a new stage of precision immunotherapy. However, CAR-T immunotherapy has been found to have many problems in clinical applications, including long treatment cycle, expensive prices, off-target effects, cytokine release syndrome, etc. Therefore, it is necessary to expand the application of CAR or adopt improved measures to enhance the therapeutic effect. This article reviews the new strategies for genetic engineering modification of CAR immune cells and the research progress and application of in situ programming to generate CAR-T, and besides, briefly introduces the new methods about the delivery of gene drugs in vivo, aiming to provide new ideas and theoretical basis for expanding and improving the application of precision immunotherapy in AML.
- acute myeloid leukemia,
- gene editing,
- chimeric antigen receptor,
- in situ programming,
- precision immunotherapy

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References

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Progress of gene-mediated precision immunotherapy in the treatment of acute myeloid leukemia