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Cell, Tissue and Gene Therapy

Section edited by David Stroncek

This section aims to improve communication between basic and clinical research related to cell, tissue and gene therapies. Effective clinical therapies require efficient translation of research between the bench and bedside to allow the discovery, development, scale up, and production of cells, tissues and vectors. These therapies are being used to treat patients with a range of diseases including cancer, cardiovascular diseases, autoimmune disorders.

Studies considered for publication include those regarding the nature of cells, tissues and vectors used in clinical therapies; methods to collect, isolate and modify cells; and novel reagents, equipment, and instruments used in production. The section also welcomes research into the application of clinical therapies; factors that effect the safety and efficacy; in particular the potency, stability and purity of cells or tissue; and issues related to the safety of cell and tissue donors.

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  1. Strict adherence to GMP guidelines and regulatory compliance is crucial when transitioning from research to clinical-grade production of ATMPs like CAR T cells. The success of CAR T cell therapy in treating he...

    Authors: Ilaria Pisani, Giusi Melita, Patricia Borges de Souza, Stefania Galimberti, Angela Maria Savino, Jolanda Sarno, Beatrice Landoni, Stefano Crippa, Elisa Gotti, Carolina Cuofano, Olga Pedrini, Chiara Capelli, Giada Matera, Daniela Belotti, Stefania Cesana, Benedetta Cabiati…
    Citation: Journal of Translational Medicine 2025 23:559
    Content type: Research Published on:

  2. B-cell maturation antigen (BCMA)-directed chimeric antigen receptor T-cell (CAR-T) therapy exhibits high response rates in patients with relapsed/refractory multiple myeloma (r/r MM). However, the specific fac...

    Authors: Ning An, Juan Li, Pan Luo, Di Wang, Peiling Zhang, Chang Shu, Songbai Cai, Qiuxia Yu, Xinyu Wen, Xinran Wang, Wei Mu, Jianlin Hu and Chunrui Li
    Citation: Journal of Translational Medicine 2025 23:552
    Content type: Research Published on:

  3. Regulatory macrophages (Mreg) represent a unique subset of macrophages known for their angiogenic and anti-inflammatory properties, positioning them as promising candidates for cell-based therapies. Recently, ...

    Authors: Karina Zitta, Rouven Berndt, Katharina Hess, Fred Fändrich, Olga Gurvich, Katja Sirviö, Tuija Kekarainen, Lars Hummitzsch, Yuk Lung Wong, Ole Sattler, Steven Braem, Mark Krebs, Axel Fudickar, Sibille Engels, Nicole Flack, Markus Steinfath…
    Citation: Journal of Translational Medicine 2025 23:551
    Content type: Research Published on:

  4. Neural stem cell (NSC) transplantation holds promising therapeutic potential for neurodegenerative disorders like amyotrophic lateral sclerosis (ALS). However, pre-clinical studies and early-phase clinical tri...

    Authors: Ivan Lombardi, Clelia Ferrero, Edvige Vulcano, Daniela Maria Rasà, Maurizio Gelati, Diego Pastor, Rose Mary Carletti, Silvia de la Morena, Daniela Celeste Profico, Sabrina Longobardi, Elisa Lazzarino, Elisa Perciballi, Jessica Diana Rosati, Salvador Martinez, Alessandro Vercelli, Angelo Luigi Vescovi…
    Citation: Journal of Translational Medicine 2025 23:529
    Content type: Research Published on:

  7. Current challenges in Chimeric Antigen Receptor (CAR) -T cell therapy for hematological cancers include T cell exhaustion and limited persistence, which contribute to cancer relapse.

    Authors: Suwan Wu, Yiyi Wei, Yingqi Qiu, Kexin Ai, Mu Chen, Hao Wang, Honghao Zhang, Qingyan Cen, Peiyun Liao, Xiangyang Ding, Xiaoling Xie and Yuhua Li
    Citation: Journal of Translational Medicine 2025 23:494
    Content type: Research Published on:

  8. Mucosal melanoma, a highly aggressive form of skin cancer, remains challenging to manage due to the lack of effective therapies. Mucin 18 (MUC18) is overexpressed in both primary and metastatic lesions of mela...

    Authors: Fenghao Zhang, Haizhen Du, Kaiping Liu, Qian Guo, Mengmeng Liang, Jing Shi, Shi Feng, Ting He, Xin-an Lu, Yanfang Tang, Lihua Wang, Qiaozhen Li, Xun Meng, Shu-Hui Liu, Yanping Ding and Yan Kong
    Citation: Journal of Translational Medicine 2025 23:473
    Content type: Research Published on:

  9. Genetic alteration of the MET receptor tyrosine kinase frequently occurs in glioblastoma (GBM). Clinically, bevacizumab treatment results in MET signaling activation, leading to GBM recurrence with a more mali...

    Authors: Anna Qin, Anna Musket, Benjamin Hilton, Johanna Preiszner, Giedre Krenciute, Michael E. Berens, Mingyao Ying, Phillip R. Musich and Qian Xie
    Citation: Journal of Translational Medicine 2025 23:460
    Content type: Research Published on:

  10. Cholangiocarcinoma (CCA) is a fatal bile duct cancer with high resistance and recurrence rates, with only one fifth of patients eligible for surgical treatment. The disease resists standard chemotherapy and of...

    Authors: Nattaporn Phanthaphol, Chalermchai Somboonpatarakun, Kwanpirom Suwanchiwasiri, Pornpimon Yuti, Jatuporn Sujjitjoon, Punn Augsornworawat, George S. Baillie, Mutita Junking and Pa-thai Yenchitsomanus
    Citation: Journal of Translational Medicine 2025 23:451
    Content type: Research Published on:

  11. The therapeutic application of chimeric antigen receptor (CAR) T cells in T-cell malignancies faces substantial limitations owing to fratricide and potential T cell aplasia, primarily attributed to the shared ...

    Authors: Haolong Lin, Lingfeng Zhang, Tong Ge, Ning An, Yongkun Yang, Yicheng Zhang and Wei Mu
    Citation: Journal of Translational Medicine 2025 23:409
    Content type: Research Published on:

  12. Despite recent advances, B cell acute lymphoblastic leukemia (B-ALL) remains a therapeutic challenge. Hematopoietic stem cell transplantation (HSCT) provides a potential cure but is hindered by various limitat...

    Authors: Wenyue Cao, Ningwen Li, Gaoxiang Wang, Hao Xu, Yang Yang, Jue Wang, Jinhuan Xu, Yun Li, Yicheng Zhang, Yang Cao and Na Wang
    Citation: Journal of Translational Medicine 2025 23:391
    Content type: Research Published on:

  13. The pathophysiological mechanism of intracranial aneurysm (IA) involves the dynamic interaction of ECM abnormalities, hemodynamic stress, and inflammatory response. The rupture of intracranial aneurysm will ca...

    Authors: Shiyu Shen, Tonglin Pan, Peixi Liu, Yanlong Tian, Yuan Shi and Wei Zhu
    Citation: Journal of Translational Medicine 2025 23:377
    Content type: Review Published on:

  14. Studies examining the therapeutic potential of Mesenchymal stem cells-derived extracellular vesicles (MSC-EVs) in wound healing and skin regeneration have progressed rapidly. Prior to considering clinical tran...

    Authors: Yufan Zhu, Han Yang, Zhixin Xue, Haojing Tang, Xihang Chen and Yunjun Liao
    Citation: Journal of Translational Medicine 2025 23:364
    Content type: Review Published on:

  16. Chimeric antigen receptor (CAR)-T cell therapy has achieved remarkable success in hematologic malignancies but faces significant limitations in gastrointestinal tumors due to the immunosuppressive tumor microe...

    Authors: Zhaorong Wu, Hongye Wang, Zhigang Zheng, Yan Lin, Linke Bian, Haigang Geng, Xiaorong Huang, Jiufei Zhu, Hongshu Jing, Yi Zhang, Chen ji and Bo Zhai
    Citation: Journal of Translational Medicine 2025 23:275
    Content type: Research Published on:

  17. Cell therapy product (CTP) developers face the significant challenge of developing appropriate potency tests for their CTPs. A review of the known potency tests used for the 31 United States Food and Drug Admi...

    Authors: Carl G. Simon Jr., Erich H. Bozenhardt, Christina M. Celluzzi, David Dobnik, Melanie L. Grant, Uma Lakshmipathy, Thiana Nebel, Linda Peltier, Anthony Ratcliffe, James L. Sherley, Glyn N. Stacey, Rouzbeh R. Taghizadeh, Eddie H. P. Tan and Sandrine Vessillier
    Citation: Journal of Translational Medicine 2025 23:259
    Content type: Review Published on:

  18. In the last two decades, many studies based on omics technologies have contributed to defining the clinical, immunological, and histological fingerprints of chronic antibody-mediated rejection (CAMR), the lead...

    Authors: Maurizio Bruschi, Simona Granata, Francesca Leone, Laura Barberio, Giovanni Candiano, Paola Pontrelli, Andrea Petretto, Martina Bartolucci, Sonia Spinelli, Loreto Gesualdo and Gianluigi Zaza
    Citation: Journal of Translational Medicine 2025 23:209
    Content type: Research Published on:

  19. Preclinical evidence suggests that young plasma has beneficial effects on multiple organ systems in aged mice. Whether young plasma exerts beneficial effects in an aging human population remains highly controv...

    Authors: Brice Gaudilliere, Lei Xue, Amy S. Tsai, Xiaoxiao Gao, Tiffany N. McAllister, Martha Tingle, Gladys Porras, Igor Feinstein, Dorien Feyaerts, Franck Verdonk, Maximilian Sabayev, Julien Hedou, Edward A. Ganio, Eloïse Berson, Martin Becker, Camilo Espinosa…
    Citation: Journal of Translational Medicine 2025 23:183
    Content type: Research Published on:

  20. Epstein-Barr virus (EBV) is an oncovirus belonging to the herpesvirus family, associated with the pathogenesis of multiple malignancies, particularly Burkitt lymphoma (BL). The virus remains latent in host cel...

    Authors: Jiajia Wang, Huiping Wang, Yangyang Ding, Nengneng Cao, Fengya Nan, Fan Wu, Cong Li, Xue Liang, Meng Xiao, Jinjing Guo, Zhimai Gao, Li Yan, Tielin Zhou, Yanli Li and Zhimin Zhai
    Citation: Journal of Translational Medicine 2025 23:171
    Content type: Research Published on:

  21. Transplant rejection remains a significant barrier to the long-term success of organ transplantation. Biopsy, although considered the gold standard, is invasive, costly, and unsuitable for routine monitoring. ...

    Authors: Yuan Song, Yihui Wang, Wenyuan Wang, Yuji Xie, Junmin Zhang, Jing Liu, Qiaofeng Jin, Wenqian Wu, He Li, Jing Wang, Li Zhang, Yali Yang, Tang Gao and Mingxing Xie
    Citation: Journal of Translational Medicine 2025 23:147
    Content type: Review Published on:

  22. Anemia is a prevalent complication of chronic kidney disease (CKD), primarily due to insufficient erythropoietin (EPO). Pegmolesatide (by Hansoh Pharma) is currently the only marketed long-acting EPO mimetic p...

    Authors: Xiaoying Ma, Zhen Li, Lu Zhang, Hui Qian, Qinkai Chen, Ye Tao, Yunfan Li, Dandan Wang, Zhizhen Hu, Weili Luo, Ping Li, Hai Yu, Changlin Mei, Xueqing Yu and Yuanfeng Zhou
    Citation: Journal of Translational Medicine 2025 23:144
    Content type: Research Published on:

  23. Chimeric antigen receptor (CAR) T-cell therapy plays a critical role in the treatment of B-cell hematologic malignancies. The combination of PD-1 inhibitors and CAR-T has shown encouraging results in treating ...

    Authors: Rui Zhang, Yi Zhang, Hairong Xiao, Qingxi Liu and Mingfeng Zhao
    Citation: Journal of Translational Medicine 2025 23:105
    Content type: Research Published on:

  24. Cutaneous melanoma is one of the most aggressive skin cancers originating from skin pigment cells. Patients with advanced melanoma suffer a poor prognosis and generally cannot benefit well from surgical resect...

    Authors: Qianrong Hu, Jiangying Xuan, Lu Wang, Kangjie Shen, Zixu Gao, Yuhong Zhou, Chuanyuan Wei and Jianying Gu
    Citation: Journal of Translational Medicine 2025 23:102
    Content type: Review Published on:

  25. T cell receptor (TCR)–engineered T cells targeting neoantigens originated from mutations in KRAS gene have demonstrated promising outcomes in clinical trials against solid tumors. However, the challenge lies in d...

    Authors: Xiaojian Han, Xiaxia Han, Yanan Hao, Bozhi Wang, Luo Li, Siyin Chen, Lin Zou, Jingjing Huang, Tong Chen, Wang Wang, Shengchun Liu, Aishun Jin and Meiying Shen
    Citation: Journal of Translational Medicine 2025 23:78
    Content type: Research Published on:

  27. Multiple myeloma (MM) is an incurable plasma cell malignancy with increasing global incidence. Chimeric antigen receptor (CAR) T-cell therapy targeting BCMA has shown efficacy in relapsed or refractory MM, but...

    Authors: Punchita Rujirachaivej, Teerapong Siriboonpiputtana, Kornkan Choomee, Kamonlapat Supimon, Thanich Sangsuwannukul, Pucharee Songprakhon, Krissada Natungnuy, Piriya Luangwattananun, Pornpimon Yuti, Mutita Junking and Pa-thai Yenchitsomanus
    Citation: Journal of Translational Medicine 2025 23:54
    Content type: Research Published on:

  28. Treatment of deep carious lesions poses significant challenges in dentistry, as complete lesion removal risks compromising pulp vitality, while selective removal often reduces the longevity of restorations. He...

    Authors: Sheetal Maria Rajan, Barsha Shrestha and Amr Fawzy
    Citation: Journal of Translational Medicine 2025 23:29
    Content type: Research Published on:

  29. In the past decades, Chimeric Antigen Receptor (CAR)-T cell therapy has achieved remarkable success, leading to the approval of six therapeutic products for haematological malignancies. Recently, the therapeut...

    Authors: E. Pinto, L. Lione, M. Compagnone, M. Paccagnella, E. Salvatori, M. Greco, V. Frezza, E. Marra, L. Aurisicchio, G. Roscilli and A. Conforti
    Citation: Journal of Translational Medicine 2025 23:10
    Content type: Review Published on:

  30. Anti-CD19 chimeric antigen receptor (CAR) T cell therapy is a common, yet highly efficient, cellular immunotherapy for lymphoma. However, many recent studies have reported on its cardiovascular (CV) toxicity. ...

    Authors: Yang Liu, Xiaoshuang Yuan, Xu Yang, Bo Yang, Guangyang Liu, Xiao Xu, Sanbin Wang, Zhixu He, Feiqing Wang and Yanju Li
    Citation: Journal of Translational Medicine 2025 23:8
    Content type: Research Published on:

  31. Hemorrhagic shock (HS) corresponds to absolute hypovolemia creating an imbalance between oxygen supply and consumption. This causes an impaired hemostasis, a systemic inflammatory response, and microvascular p...

    Authors: Nathalie Baudry, Aurélie Campeanu, Clotilde Aussel, Caroline Doutrelon, Marion Grosbot, Sébastien Banzet, Eric Vicaut and Juliette Peltzer
    Citation: Journal of Translational Medicine 2024 22:1143
    Content type: Research Published on:

  32. Immunotherapy utilizing dendritic cells (DCs) and cytokine-induced killer (CIK) cells is a promising treatment approach for solid tumors. This systematic review and meta-analysis aimed to evaluate the efficacy...

    Authors: Wendi Jiang, Zhongda Wang, Qinghuizi Luo, Zhe Dai, Jialong Zhu, Xiaoyue Tao, Yiyang Xie, Yuanyang Du, Longwei Jiang, Xiaoyuan Chu, Gongbo Fu and Zengjie Lei
    Citation: Journal of Translational Medicine 2024 22:1122
    Content type: Research Published on:

  33. Colorectal cancer (CRC) is the third most common cancer worldwide, with highly variable prognosis and response to treatment. A large subset of patients does not respond to standard treatments or develops resis...

    Authors: Erica Torchiaro, Marco Cortese, Consalvo Petti, Marco Basirico’, Federica Invrea, Alice D’Andrea, Letizia Franco, Dario Sangiolo and Enzo Medico
    Citation: Journal of Translational Medicine 2024 22:1100
    Content type: Research Published on:

  34. B-cell maturation antigen (BCMA)-targeted chimeric antigen receptor T-cell (CAR T-cell) therapy has exhibited remarkable efficacy in refractory or relapsed multiple myeloma (R/R MM), but recurrence and rapid p...

    Authors: Yuhan Yan, Yixuan Tu, Qian Cheng, Jian Zhang, Erhua Wang, Zuqun Deng, Yan Yu, Liwen Wang, Rui Liu, Ling Chu, Liqing Kang, Jing Liu and Xin Li
    Citation: Journal of Translational Medicine 2024 22:1087
    Content type: Research Published on:

  35. Over the past decade, chimeric antigen receptor (CAR)-T cell therapy has emerged as a revolutionary immunotherapeutic approach to combat cancer. This therapy constructs a CAR on the surface of T cells through ...

    Authors: Yan Liu, Lin Xiao, Mingxuan Yang, Xuemei Chen, Hongyue Liu, Quanxing Wang, Meng Guo and Jianhua Luo
    Citation: Journal of Translational Medicine 2024 22:1076
    Content type: Review Published on:

  36. Ion channels in retinal pigment epithelial (RPE) cells are crucial for retinal health and vision functions. Defects in such channels are intricately associated with the development of various retinopathies tha...

    Authors: Ping Xu, Weisheng Zou, Wenjing Yin, Guifu Chen, Guanjie Gao and Xiufeng Zhong
    Citation: Journal of Translational Medicine 2024 22:1073
    Content type: Review Published on:

  38. Improved treatment options are urgently needed for neurological injuries resulting from trauma or iatrogenic events causing long-term disabilities that severely impact patients’ quality of life. In vitro and a...

    Authors: Xiujuan Wang, Qian Wang, Ziyao Xia, Ying Yang, Xunan Dai, Chun Zhang, Jiaxian Wang and Yongsheng Xu
    Citation: Journal of Translational Medicine 2024 22:1055
    Content type: Review Published on:

  39. Chimeric antigen receptor T-cell (CAR-T) immunotherapy has achieved remarkable success in treating chemotherapy-refractory hematological malignancies. However, its efficacy in solid and brain tumors remains li...

    Authors: Yuqing Luo, Jianqiao Shentu, Hening Xu, Yongming Xia, Lili Fang and Shiwei Duan
    Citation: Journal of Translational Medicine 2024 22:1019
    Content type: Commentary Published on:

  41. Meningeal metastasis (LM) is commonly seen in the advanced stages of cancer patients, often leading to a rapid decline in survival time and quality of life. Currently, there is still a lack of standardized tre...

    Authors: Jia-Li Zhao, Bi-Lin Lin, Chen Luo, Yan-ling Yi, Peng Huang, Yu Chen, Sha Zhao, Zhen-Jie Huang, Xin-Yi Ma and Long Huang
    Citation: Journal of Translational Medicine 2024 22:1000
    Content type: Review Published on:

  42. Diabetic vascular complications attract increased attention due to their high morbidity, mortality and disability rate. Comprehensive and in-depth exploration of the etiology and pathogenesis of diabetic vascu...

    Authors: Xinyi Fang, Yuxin Zhang, Yanjiao Zhang, Huifang Guan, Xinyue Huang, Runyu Miao, Ruiyang Yin and Jiaxing Tian
    Citation: Journal of Translational Medicine 2024 22:944
    Content type: Review Published on:

  43. Breast cancer (BC) is the most common malignancy in women. Immunotherapy has revolutionized treatment options in many malignancies, and the introduction of immune checkpoint inhibition yielded beneficial resul...

    Authors: Ilona Hagelstein, Laura Wessling, Alexander Rochwarger, Latifa Zekri, Boris Klimovich, Christian M. Tegeler, Gundram Jung, Christian M. Schürch, Helmut R. Salih and Martina S. Lutz
    Citation: Journal of Translational Medicine 2024 22:902
    Content type: Research Published on:

  44. TCR-T cell therapy represents a promising advancement in adoptive immunotherapy for cancer treatment. Despite its potential, the development and preclinical testing of TCR-T cells face significant challenges. ...

    Authors: Elena A. Golikova, Alina A. Alshevskaya, Saleh Alrhmoun, Natalia A. Sivitskaya and Sergey V. Sennikov
    Citation: Journal of Translational Medicine 2024 22:897
    Content type: Review Published on:

    The Correction to this article has been published in Journal of Translational Medicine 2024 22:1067

  45. The incidence of extramedullary diseases (EMDs) in patients diagnosed with acute myeloid leukemia (AML) is approximately 10–20%. These patients exhibit a significantly distinct etiology, therapeutic response, ...

    Authors: Yifan Zhao, Xue Bai, Shujing Guo, Xiaomei Zhang, Jile Liu, Mohan Zhao, Tianle Xie, Haotian Meng, Yu Zhang, Xiaoyuan He and Mingfeng Zhao
    Citation: Journal of Translational Medicine 2024 22:888
    Content type: Research Published on:

  46. Recombinant adeno-associated viruses (rAAVs) have emerged as an attractive tool for gene delivery, and demonstrated tremendous promise in gene therapy and gene editing—therapeutic modalities with potential “on...

    Authors: Liang Xu, Shun Yao, Yifan Evan Ding, Mengxiao Xie, Dingqi Feng, Pengfei Sha, Lu Tan, Fengfeng Bei and Yizheng Yao
    Citation: Journal of Translational Medicine 2024 22:866
    Content type: Review Published on:

  47. Glioblastoma (GBM) is the most frequent and aggressive brain tumor in adults with the lowest survival rates five years post-diagnosis. Oncolytic viruses (OVs) selectively target and damage cancer cells, and fo...

    Authors: Alberto Reale, Andrea Gatta, Amruth Kaleem Basha Shaik, Mariam Shallak, Anna Maria Chiaravalli, Michele Cerati, Martina Zaccaria, Stefano La Rosa, Arianna Calistri, Roberto Sergio Accolla and Greta Forlani
    Citation: Journal of Translational Medicine 2024 22:862
    Content type: Research Published on:

  48. Highly efficient adeno associated viruses (AAVs) targeting the central nervous system (CNS) are needed to deliver safe and effective therapies for inherited neurological disorders. The goal of this study was t...

    Authors: Monika Chauhan, Audrey L. Daugherty, Fatemeh (Ellie) Khadir, Ozgun F. Duzenli, Alexandra Hoffman, Jennifer A. Tinklenberg, Peter B. Kang, George Aslanidi and Christina A. Pacak
    Citation: Journal of Translational Medicine 2024 22:824
    Content type: Research Published on:

  49. Kidneys are at risk from drug-induced toxicity, with a significant proportion of acute kidney injury (AKI) linked to medications, particularly cisplatin. Existing cytoprotective drugs for cisplatin-AKI carry s...

    Authors: Abantika Ganguly, Shashank Chetty, Rosita Primavera, Steven Levitte, Shobha Regmi, Benjamin William Dulken, Scott M. Sutherland, Wendy Angeles, Jing Wang and Avnesh S. Thakor
    Citation: Journal of Translational Medicine 2024 22:789
    Content type: Research Published on:

  50. The effects of mesenchymal stem cells (MSCs) on heart failure (HF) have been controversial. This study was conducted to investigate whether the transplantation of MSCs after HF could help improve clinical outc...

    Authors: Shahin Kavousi, Alireza Hosseinpour, Fateme Bahmanzadegan Jahromi and Armin Attar
    Citation: Journal of Translational Medicine 2024 22:786
    Content type: Research Published on:
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