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Recent advances in lentiviral vectors for gene therapy

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Abstract

Lentiviral vectors (LVs), derived from human immunodeficiency virus, are powerful tools for modifying the genes of eukaryotic cells such as hematopoietic stem cells and neural cells. With the extensive and in-depth studies on this gene therapy vehicle over the past two decades, LVs have been widely used in both research and clinical trials. For instance, third-generation and self-inactive LVs have been used to introduce a gene with therapeutic potential into the host genome and achieve targeted delivery into specific tissue. When LVs are employed in leukemia, the transduced T cells recognize and kill the tumor B cells; in β-thalassemia, the transduced CD34+ cells express normal β-globin; in adenosine deaminase-deficient severe combined immunodeficiency, the autologous CD34+ cells express adenosine deaminase and realize immune reconstitution. Overall, LVs can perform significant roles in the treatment of primary immunodeficiency diseases, hemoglobinopathies, B cell leukemia, and neurodegenerative diseases. In this review, we discuss the recent developments and therapeutic applications of LVs. The safe and efficient LVs show great promise as a tool for human gene therapy.

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Acknowledgements

This work was supported by the National Key Research and Development Program of China (2020YFC2008302), the Sichuan Science and Technology program (2019YFG0266), and the 1.3.5 project for disciplines of excellence, West China Hospital, Sichuan University (ZYJC18028, 2021HXFH064). All the figures in this review were created with BioRender.com.

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Authors and Affiliations

  1. Department of Pharmacy, State Key Laboratory of Biotherapy and Cancer Center, National Clinical Research Center for Geriatrics, West China Hospital, Sichuan University, Chengdu, 610041, China

    Xiaoyu Wang, Cuicui Ma, Zhou Qin, Ting Xu, Zhiyao He & Yuquan Wei

  2. Department Clinical Neurophysiology, Centre for the Research and Rehabilitation of Hereditary Ataxias, Holguín, 80100, Cuba

    Roberto Rodríguez Labrada

  3. Key Laboratory of Drug-Targeting and Drug Delivery System of the Education Ministry, Sichuan Engineering Laboratory for Plant-Sourced Drug and Sichuan Research Center for Drug Precision Industrial Technology, West China School of Pharmacy, Sichuan University, Chengdu, 610041, China

    Ting Xu & Zhiyao He

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Wang, X., Ma, C., Rodríguez Labrada, R. et al. Recent advances in lentiviral vectors for gene therapy. Sci. China Life Sci. 64, 1842–1857 (2021). https://doi.org/10.1007/s11427-021-1952-5

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