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Journal Articles

Analysis of efficacy and prognostic factors in transfusion-dependent thalassemia following allogeneic hematopoietic stem cell transplantation using the ‘gx-07-tm’ regimen

Blood (2025) 146 (Supplement 1): 167.

https://doi.org/10.1182/blood-2025-167

Published: 2025

Journal Articles

Daclizumab enhances aGVHD prophylaxis by modulating T-cell polarization while preserving the graft-versus-lymphoma effect

Yanfei Lu, Gaohui Yang, Min Wu, Yongrong Lai, Lingling Shi, Rongrong Liu

Blood (2025) 146 (Supplement 1): 7611.

https://doi.org/10.1182/blood-2025-7611

Published: 2025

Journal Articles

Clinical characteristics and transplant outcomes in pediatric patients with transfusion-dependent alpha-thalassemia undergoing HSCT

Lihua Huang, Lingling Shi, Zhenbin Wei, Gaohui Yang, Lianjin Liu, Zhongming Zhang, Hongwen Xiao, Lingyuan Pan, Wenqiang Xie, Zhaoping Gan, Beicai Liu, Xuemei Zhou, Yinghua Chen, Yanye Liu, Yongrong Lai, Rongrong Liu

Blood (2025) 146 (Supplement 1): 7826.

https://doi.org/10.1182/blood-2025-7826

Published: 2025

Journal Articles

Development and validation of a machine Learning–Based clinical prediction model for hemorrhagic cystitis after allogeneic hematopoietic stem cell transplantation in thalassemia major

Zhaoping Gan, Zhenbin Wei, Gaohui Yang, Yumei Huang, Lingyuan Pan, Lingling Shi, Soulivanh Nanthachak, Cailan Wen, Lianjin Liu, Yongrong Lai, Rongrong Liu

Blood (2025) 146 (Supplement 1): 7874.

https://doi.org/10.1182/blood-2025-7874

Published: 2025

Journal Articles

All 19 subjects with transfusion-dependent β-thalassemia achieved transfusion independence after treatment with RM-001 (autologous HBG1/2 promoter-modified CD34+ hematopoietic stem and progenitor cells)

Rongrong Liu, Li Wang, Hui Xu, Jianpei Fang, Sixi Liu, Xiaolin Yin, Junbin Liang, Xinyu Li, Yue Li, Gaohui Yang, Yaoyun Li, Yali Zhou, Lin Cheng, Yongrong Lai, Junjiu Huang, Xinhua Zhang

Blood (2025) 146 (Supplement 1): 4308.

https://doi.org/10.1182/blood-2025-4308

Published: 2025

Journal Articles

Multimodal evaluation of brain injury and cerebral iron deposition in thalassemia patients: A combined human-imaging and murine model study

Jingxuan Shao, Cheng Tang, Yanfei Lu, Min Wu, Zhenbin Wei, Zhaoping Gan, Yumei Huang, Peng Peng, Cailan Wen, Yongrong Lai, Rongrong Liu

Blood (2025) 146 (Supplement 1): 2917.

https://doi.org/10.1182/blood-2025-2917

Published: 2025

Journal Articles

Snh-119014, a novel pyruvate kinase activator, enhances ATP production and reduces oxidative stress in erythroid cells from patients with β-thalassemia major

Huang Qiulin, Yumei Huang, Lingyuan Pan, Zhaoping Gan, Soulivanh Nanthachak, Rongrong Liu, Yongrong Lai

Blood (2025) 146 (Supplement 1): 4698.

https://doi.org/10.1182/blood-2025-4698

Published: 2025

Journal Articles

A phase 2 study of luspatercept in adults and adolescents with α-thalassemia: Findings from the dose-confirmation cohort in adolescents

Yongrong Lai, Meng-Yao Lu, Ying Zhao, Antonis Kattamis, Gian Luca Forni, Kitty Formentin, Pablo Gurman, Mingzhao Gao, Loyse Felber Medlin, Patricia Martin-Regueira, Vincent Jaquet, Vip Viprakasit

Blood (2025) 146 (Supplement 1): 4689.

https://doi.org/10.1182/blood-2025-4689

Published: 2025

Journal Articles

Iron overload disrupts alveolar-capillary tight junctions via oxidative stress in a murine model

Xiao Liang, Gaohui Yang, Yumei Huang, Lingling Shi, Lingyuan Pan, Zhaoping Gan, Zhenbin Wei, Yanfei Lu, Min Wu, Cailan Wen, Jingxuan Shao, Yongrong Lai, Rongrong Liu

Blood (2025) 146 (Supplement 1): 4683.

https://doi.org/10.1182/blood-2025-4683

Published: 2025

Journal Articles

Rapid, efficient and durable fetal hemoglobin production following CS-101 treatment in transfusion-dependent β-thalassemia participants: An autologous, ex vivo edited CD34+ stem cell product using the innovative transformer base editor (tBE)

Jia Chen, Yongrong Lai, Xiaowen Zhai, Wei Li Zhao, Rongrong Liu, Xiaowen Qian, Wei Tang, Lijie Wang, Xu-Kai Ma, Yaliang Li, Jia Wei, Kewei Lan, Yan Zhang, Li Yang, Xiaodun (Susan) Mou

Blood (2025) 146 (Supplement 1): 6090.

https://doi.org/10.1182/blood-2025-6090

Published: 2025

Journal Articles

Sirolimus plus low-exposure calcineurin inhibitors as an alternative gvhd prophylaxis in CNI-intolerant recipients: A retrospective study

Min Wu, Yanfei Lu, Zhenbin Wei, Lingling Shi, Gaohui Yang, Rui Huang, Na Sun, Zhaoping Gan, Huicheng Huang, Xuemei Zhou, Lianjin Liu, Zhongming Zhang, Yongrong Lai, Rongrong Liu

Blood (2025) 146 (Supplement 1): 7746.

https://doi.org/10.1182/blood-2025-7746

Published: 2025

Journal Articles

Addition of Daclizumab to Standard Graft-Versus-Host Disease Prophylaxis in Patients with Thalassemia Major Undergoing Transplantation from Alternative Donor Hematopoietic Stem-Cell Transplantation: A Single-Center Retrospective Analysis from China

Blood (2024) 144 (Supplement 1): 2152.

https://doi.org/10.1182/blood-2024-205021

Published: 2024

Journal Articles

Pulmonary Dysfunction in Patients with Transfusion-Dependent Thalassemia

Xiao Liang, Gaohui Yang, Lingling Shi, Lianjin Liu, Zhenbin Wei, Yumei Huang, Jing Bai, Yi Liang, Lingyuan Pan, Zhaoping Gan, Yu Lin, Yibin Yao, Ying Tang, Huicheng Huang, Xuemei Zhou, Qifa Liu, Yongrong Lai, Rongrong Liu

Blood (2024) 144 (Supplement 1): 1096.

https://doi.org/10.1182/blood-2024-204971

Published: 2024

Journal Articles

Using T-Lymphocyte Subsets at Engraftment to Predict the Risk of Acute Graft Versus Host Disease in Patients with Thalassemia Major：Development of a New Predictive Nomogram

Hongwen Xiao, Gaohui Yang, Qiuling Huang, Zhenbin Wei, Zhaoping Gan, Meiqing Wu, Zeyan Shi, Huicheng Huang, Zhaofang Pan, Lianjin Liu, Lingling Shi, Zhongming Zhang, Qifa Liu, Rongrong Liu, Yongrong Lai

Blood (2024) 144 (Supplement 1): 2139.

https://doi.org/10.1182/blood-2024-205258

Published: 2024

Journal Articles

The Optimal Dose of Anti-Thymocyte Globulin in Beta-Thalassemia Major Following Matched Sibling Donor Hematopoietic Stem Cell Transplantation: A Single-Center, Open-Label, Randomized, Controlled Clinical Study

Qiuling Huang, Rongrong Liu, Lianjin Liu, Meiqing Wu, Lingling Shi, Gaohui Yang, Zhongming Zhang, Qifa Liu, Yongrong Lai

Blood (2024) 144 (Supplement 1): 2134.

https://doi.org/10.1182/blood-2024-205204

Published: 2024

Journal Articles

A Multi-Center Clinical Trial of Allogeneic Hematopoietic Stem Cell Transplants in Transfusion Dependent Thalassemia

Blood (2024) 144 (Supplement 1): 378.

https://doi.org/10.1182/blood-2024-204984

Published: 2024

Journal Articles

Rapid, Efficient and Durable Fetal Hemoglobin Production Following CS-101 Treatment in Transfusion-Dependent β-Thalassemia Participants: An Autologous, Ex Vivo Edited CD34+ Stem Cell Product Using the Innovative Transformer Base Editor (tBE)

Yongrong Lai, Rongrong Liu, Lijie Wang, Xu-Kai Ma, Yaliang Li, Gaohui Yang, Lingling Shi, Jia Wei, Zhenbin Wei, Xuemei Zhou, Kewei Lan, Yan Zhang, Xiaodun (Susan) Mou, Li Yang, Jia Chen

Blood (2024) 144 (Supplement 1): 4962.

https://doi.org/10.1182/blood-2024-200691

Published: 2024

Journal Articles

Updated Safety and Efficacy Results of RM-001, Autologous HBG1/2 Promoter-Modified CD34+ Hematopoietic Stem and Progenitor Cells, in Treating Transfusion-Dependent β-Thalassemia

Rongrong Liu, Li Wang, Hui Xu, Jianpei Fang, Sixi Liu, Xiaolin Yin, Junbin Liang, Xinyu Li, Yue Li, Gaohui Yang, Yaoyun Li, Yali Zhou, Lei Shi, Yongrong Lai, Junjiu Huang, Xinhua Zhang

Blood (2024) 144 (Supplement 1): 4960.

https://doi.org/10.1182/blood-2024-206549

Published: 2024

Journal Articles

Oxidative Stress Damage of Iron Overload on Bone Marrow Erythropoiesis, Heart and Liver in Non-Transfusion Dependent Thalassemia

Lin Pan, Yanni Xie, Xianqing Yin, Yumei Huang, Gaohui Yang, Chan Li, Yongyu Chen, Fengling Liu, Ning Zhang, Xiao Liang, Zhaoping Gan, Zhenbin Wei, Haiyan Liang, Qifa Liu, Yongrong Lai, Rongrong Liu

Blood (2023) 142 (Supplement 1): 5245.

https://doi.org/10.1182/blood-2023-180898

Published: 2023

Journal Articles

Safety and Efficacy of RM-001 (Autologous HBG1/2 Promoter-modified CD34+ Hematopoietic Stem and Progenitor Cells) in Patients with Transfusion-Dependent β-Thalassemia

Rongrong Liu, Li Wang, Hui Xu, Xiaolin Yin, Junbin Liang, Wenqiang Xie, Gaohui Yang, Yaoyun Li, Yali Zhou, Lei Shi, Bin Xiao, Lingling Shi, Zeyan Shi, Xuemei Zhou, Xiangmin Xu, Jianpei Fang, Yongrong Lai, Junjiu Huang, Xinhua Zhang

Blood (2023) 142 (Supplement 1): 4994.

https://doi.org/10.1182/blood-2023-181453

Published: 2023

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Analysis of efficacy and prognostic factors in transfusion-dependent thalassemia following allogeneic hematopoietic stem cell transplantation using the ‘gx-07-tm’ regimen

Daclizumab enhances aGVHD prophylaxis by modulating T-cell polarization while preserving the graft-versus-lymphoma effect

Clinical characteristics and transplant outcomes in pediatric patients with transfusion-dependent alpha-thalassemia undergoing HSCT

Development and validation of a machine Learning–Based clinical prediction model for hemorrhagic cystitis after allogeneic hematopoietic stem cell transplantation in thalassemia major

All 19 subjects with transfusion-dependent β-thalassemia achieved transfusion independence after treatment with RM-001 (autologous HBG1/2 promoter-modified CD34+ hematopoietic stem and progenitor cells)

Multimodal evaluation of brain injury and cerebral iron deposition in thalassemia patients: A combined human-imaging and murine model study

Snh-119014, a novel pyruvate kinase activator, enhances ATP production and reduces oxidative stress in erythroid cells from patients with β-thalassemia major

A phase 2 study of luspatercept in adults and adolescents with α-thalassemia: Findings from the dose-confirmation cohort in adolescents

Iron overload disrupts alveolar-capillary tight junctions via oxidative stress in a murine model

Rapid, efficient and durable fetal hemoglobin production following CS-101 treatment in transfusion-dependent β-thalassemia participants: An autologous, ex vivo edited CD34+ stem cell product using the innovative transformer base editor (tBE)

Sirolimus plus low-exposure calcineurin inhibitors as an alternative gvhd prophylaxis in CNI-intolerant recipients: A retrospective study

Addition of Daclizumab to Standard Graft-Versus-Host Disease Prophylaxis in Patients with Thalassemia Major Undergoing Transplantation from Alternative Donor Hematopoietic Stem-Cell Transplantation: A Single-Center Retrospective Analysis from China

Pulmonary Dysfunction in Patients with Transfusion-Dependent Thalassemia

Using T-Lymphocyte Subsets at Engraftment to Predict the Risk of Acute Graft Versus Host Disease in Patients with Thalassemia Major：Development of a New Predictive Nomogram

The Optimal Dose of Anti-Thymocyte Globulin in Beta-Thalassemia Major Following Matched Sibling Donor Hematopoietic Stem Cell Transplantation: A Single-Center, Open-Label, Randomized, Controlled Clinical Study

A Multi-Center Clinical Trial of Allogeneic Hematopoietic Stem Cell Transplants in Transfusion Dependent Thalassemia

Rapid, Efficient and Durable Fetal Hemoglobin Production Following CS-101 Treatment in Transfusion-Dependent β-Thalassemia Participants: An Autologous, Ex Vivo Edited CD34+ Stem Cell Product Using the Innovative Transformer Base Editor (tBE)

Updated Safety and Efficacy Results of RM-001, Autologous HBG1/2 Promoter-Modified CD34+ Hematopoietic Stem and Progenitor Cells, in Treating Transfusion-Dependent β-Thalassemia

Oxidative Stress Damage of Iron Overload on Bone Marrow Erythropoiesis, Heart and Liver in Non-Transfusion Dependent Thalassemia

Safety and Efficacy of RM-001 (Autologous HBG1/2 Promoter-modified CD34+ Hematopoietic Stem and Progenitor Cells) in Patients with Transfusion-Dependent β-Thalassemia

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