Gene therapy and gene editing strategies in inherited blood disorders

Xuemei Song; JinLei Liu; Tangcong Chen; Tingfeng Zheng; Xiaolong Wang; Xiang Guo

doi:10.1016/j.jgg.2024.07.004

Volume 51 Issue 11

Nov. 2024

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Article Navigation > Journal of Genetics and Genomics > 2024 > 51(11): 1162-1172

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Gene therapy and gene editing strategies in inherited blood disorders

doi: 10.1016/j.jgg.2024.07.004

Institute of Blood Diseases, Department of Hematology, Sichuan Academy of Medical Sciences & Sichuan Provincial People's Hospital, School of Medicine of University of Electronic Science and Technology of China, Chengdu, Sichuan 610000, China

Funds:

This work was supported by Research Fund of Sichuan Academy of Medical Sciences and Sichuan Provincial People's Hospital (30420230006), the National Natural Science Foundation of China (Nos. 82300142), and Sichuan Science and Technology Program (2022ZYD0131).

Received Date: 2024-04-30
Accepted Date: 2024-07-02
Rev Recd Date: 2024-07-01

Available Online: 2025-06-06

Publish Date: 2024-07-08

Abstract

Abstract

Gene therapy has shown significant potential in treating various diseases, particularly inherited blood disorders such as hemophilia, sickle cell disease, and thalassemia. Advances in understanding the regulatory network of disease-associated genes have led to the identification of additional therapeutic targets for treatment, especially for β-hemoglobinopathies. Erythroid regulatory factor BCL11A offers the most promising therapeutic target for β-hemoglobinopathies, and reduction of its expression using the commercialized gene therapy product Casgevy has been approved for use in the UK and USA in 2023. Notably, the emergence of innovative gene editing technologies has further broadened the gene therapy landscape, presenting possibilities for treatment. Intensive studies indicate that base editing and prime editing, built upon CRISPR technology, enable precise single-base modification in hematopoietic stem cells for addressing inherited blood disorders ex vivo and in vivo. In this review, we present an overview of the current landscape of gene therapies, focusing on clinical research and gene therapy products for inherited blood disorders, evaluation of potential gene targets, and the gene editing tools employed in current gene therapy practices, which provides an insight for the establishment of safer and more effective gene therapy methods for a wider range of diseases in the future.
- Gene therapy,
- Inherited blood disorders,
- Therapeutic targets,
- β-Hemoglobinopathies,
- Gene editing

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References(106)

References

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