Research progress and considerations for thalassemia gene therapy
Traditional treatment modalities for thalassemia include regular blood transfusions and allogenic hematopoietic stem cell transplantation (allo-HSCT). In recent years, autologous transplantation of gene-modified hematopoietic stem cells has emerged as a new curative strategy for transfusion-dependen...
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Editorial Office of Journal of Shanghai Jiao Tong University (Medical Science)
2025-05-01
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| Series: | Shanghai Jiaotong Daxue xuebao. Yixue ban |
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| Online Access: | https://xuebao.shsmu.edu.cn/article/2025/1674-8115/1674-8115-2025-45-5-540.shtml |
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| _version_ | 1850219214614298624 |
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| author | GAO Xinjie LIU Yan WANG Dawei |
| author_facet | GAO Xinjie LIU Yan WANG Dawei |
| author_sort | GAO Xinjie |
| collection | DOAJ |
| description | Traditional treatment modalities for thalassemia include regular blood transfusions and allogenic hematopoietic stem cell transplantation (allo-HSCT). In recent years, autologous transplantation of gene-modified hematopoietic stem cells has emerged as a new curative strategy for transfusion-dependent thalassemia (TDT),which has the potential to replace conventional treatments, and provide lifelong benefits for patients. There are two existing technical approaches for gene therapy of β-thalassemia: gene addition, which involves transducing exogenous β-globin genes into hematopoietic stem cells (HSCs), and gene editing, which utilizes CRISPR-Cas9 or other editing systems to re-activate the expression of γ-globin gene. This article summarizes the marketed products and research progress in clinical trials, aiming to analyze the respective advantages and limitations of these two approaches, and discusses the effectiveness and safety of current gene therapies for β-thalassemia, as well as the future directions for associated technologies, including ex vivo HSC expansion with maintenance of stemness and vector-mediated in vivo gene modification. In terms of clinical translational medicine, this article provides in-depth insights into promising solutions for contemporary challenges confronted in clinical trials, including process development challenges, clinical trial conduct, regulatory approval processes, commercialization and payment systems. |
| format | Article |
| id | doaj-art-c1a9a4de0a214a61aef7f43641980621 |
| institution | OA Journals |
| issn | 1674-8115 |
| language | zho |
| publishDate | 2025-05-01 |
| publisher | Editorial Office of Journal of Shanghai Jiao Tong University (Medical Science) |
| record_format | Article |
| series | Shanghai Jiaotong Daxue xuebao. Yixue ban |
| spelling | doaj-art-c1a9a4de0a214a61aef7f436419806212025-08-20T02:07:27ZzhoEditorial Office of Journal of Shanghai Jiao Tong University (Medical Science)Shanghai Jiaotong Daxue xuebao. Yixue ban1674-81152025-05-0145554054810.3969/j.issn.1674-8115.2025.05.0021674-8115（2025）05-0540-09Research progress and considerations for thalassemia gene therapyGAO Xinjie0LIU Yan1WANG Dawei2Shanghai Institute of Hematology, State Key Laboratory of Medical Genomics, National Research Center for Translational Medicine (Shanghai), Ruijin Hospital, Shanghai Jiao Tong University School of Medicine, Shanghai 200025, ChinaShanghai Institute of Hematology, State Key Laboratory of Medical Genomics, National Research Center for Translational Medicine (Shanghai), Ruijin Hospital, Shanghai Jiao Tong University School of Medicine, Shanghai 200025, ChinaShanghai Institute of Hematology, State Key Laboratory of Medical Genomics, National Research Center for Translational Medicine (Shanghai), Ruijin Hospital, Shanghai Jiao Tong University School of Medicine, Shanghai 200025, ChinaTraditional treatment modalities for thalassemia include regular blood transfusions and allogenic hematopoietic stem cell transplantation (allo-HSCT). In recent years, autologous transplantation of gene-modified hematopoietic stem cells has emerged as a new curative strategy for transfusion-dependent thalassemia (TDT),which has the potential to replace conventional treatments, and provide lifelong benefits for patients. There are two existing technical approaches for gene therapy of β-thalassemia: gene addition, which involves transducing exogenous β-globin genes into hematopoietic stem cells (HSCs), and gene editing, which utilizes CRISPR-Cas9 or other editing systems to re-activate the expression of γ-globin gene. This article summarizes the marketed products and research progress in clinical trials, aiming to analyze the respective advantages and limitations of these two approaches, and discusses the effectiveness and safety of current gene therapies for β-thalassemia, as well as the future directions for associated technologies, including ex vivo HSC expansion with maintenance of stemness and vector-mediated in vivo gene modification. In terms of clinical translational medicine, this article provides in-depth insights into promising solutions for contemporary challenges confronted in clinical trials, including process development challenges, clinical trial conduct, regulatory approval processes, commercialization and payment systems.https://xuebao.shsmu.edu.cn/article/2025/1674-8115/1674-8115-2025-45-5-540.shtmlthalassemiagene therapyautologous hematopoietic stem-cell transplantationgene editingtranslational medicine |
| spellingShingle | GAO Xinjie LIU Yan WANG Dawei Research progress and considerations for thalassemia gene therapy Shanghai Jiaotong Daxue xuebao. Yixue ban thalassemia gene therapy autologous hematopoietic stem-cell transplantation gene editing translational medicine |
| title | Research progress and considerations for thalassemia gene therapy |
| title_full | Research progress and considerations for thalassemia gene therapy |
| title_fullStr | Research progress and considerations for thalassemia gene therapy |
| title_full_unstemmed | Research progress and considerations for thalassemia gene therapy |
| title_short | Research progress and considerations for thalassemia gene therapy |
| title_sort | research progress and considerations for thalassemia gene therapy |
| topic | thalassemia gene therapy autologous hematopoietic stem-cell transplantation gene editing translational medicine |
| url | https://xuebao.shsmu.edu.cn/article/2025/1674-8115/1674-8115-2025-45-5-540.shtml |
| work_keys_str_mv | AT gaoxinjie researchprogressandconsiderationsforthalassemiagenetherapy AT liuyan researchprogressandconsiderationsforthalassemiagenetherapy AT wangdawei researchprogressandconsiderationsforthalassemiagenetherapy |