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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.

Featured articles

Phase I and registry study of autologous bone marrow concentrate evaluated in PDE5 inhibitor refractory erectile dysfunction

Research | Mark Bieri et al


Rationale for the clinical use of adipose-derived mesenchymal stem cells for COVID-19 patients​​​​​​​

Review | Christopher J. Rogers et al


Allo-priming as a universal anti-viral vaccine: protecting elderly from current COVID-19 and any future unknown viral outbreak

Review |  Michael Har-Noy & Reuven Or 

Page 1 of 11

  1. Chimeric antigen receptor T (CAR-T) cell therapy, as an emerging anti-tumor treatment, has garnered extensive attention in the study of targeted therapy of multiple tumor-associated antigens in hepatocellular ...

    Authors: Keming Lin, Baijin Xia, Xuemei Wang, Xin He, Mo Zhou, Yingtong Lin, Yidan Qiao, Rong Li, Qier Chen, Yuzhuang Li, Jinzhu Feng, Tao Chen, Cancan Chen, Xinyu Li, Hui Zhang, Lijuan Lu…
    Citation: Journal of Translational Medicine 2024 22:349
  2. The shortage of organs for transplantation emphasizes the urgent need for alternative solutions. Xenotransplantation has emerged as a promising option due to the greater availability of donor organs. However, ...

    Authors: Yinglin Yuan, Yuanyuan Cui, Dayue Zhao, Yuan Yuan, Yanshuang Zhao, Danni Li, Xiaomei Jiang and Gaoping Zhao
    Citation: Journal of Translational Medicine 2024 22:324
  3. Rheumatoid arthritis (RA) is an autoimmune disease characterized by inflammatory reactions and tissue damage in the joints. Long-term drug use in clinical practice is often accompanied by adverse reactions. Ex...

    Authors: Yuwei Lin, Zhanrui Cheng, Yan Zhong, Yinting Zhao, Guifen Xiang, Ling Li, Li Tian and Zhong Liu
    Citation: Journal of Translational Medicine 2024 22:305
  4. CLAD (Chronic Lung Allograft Dysfunction) remains a serious complication following lung transplantation. Some evidence shows that portions of Extracorporeal Photopheresis (ECP)-treated patients improve/stabili...

    Authors: Sara Bozzini, Eleonora Bozza, Cecilia Bagnera, Claudia Del Fante, Eugenio Barone, Simona De Vitis, Mara De Amici, Giorgia Testa, Stefania Croce, Chiara Valsecchi, Maria A Avanzini, Rosalia Cacciatore, Cristina Mortellaro, Gianluca Viarengo, Cesare Perotti and Federica Meloni
    Citation: Journal of Translational Medicine 2024 22:276
  5. The prognostic significance of myelofibrosis (MF) grade in patients with myelodysplastic syndrome (MDS) following an allogeneic hematopoietic stem cell transplantation (allo-HSCT) remains elusive.

    Authors: Panpan Zhu, Xiaoyu Lai, Lizhen Liu, Jimin Shi, Jian Yu, Yanmin Zhao, Luxin Yang, Tingting Yang, Weiyan Zheng, Jie Sun, Wenjun Wu, Yi Zhao, Zhen Cai, He Huang and Yi Luo
    Citation: Journal of Translational Medicine 2024 22:275
  6. Chimeric antigen receptor natural killer (CAR-NK) cells represent a promising advancement in CAR cell therapy, addressing limitations observed in CAR-T cell therapy. However, our prior study revealed challenge...

    Authors: Na Yang, Caili Zhang, Yingchun Zhang, Yuting Fan, Jing Zhang, Xiaojin Lin, Ting Guo, Yangzuo Gu, Jieheng Wu, Jianmei Gao, Xing Zhao and Zhixu He
    Citation: Journal of Translational Medicine 2024 22:274
  7. Chimeric antigen receptor (CAR) T cell therapy is associated with high risk of adverse events. Glucocorticoids (GCs) are cornerstone in the management of high-grade cytokine release syndrome (CRS) and immune e...

    Authors: Thomas Poiret, Sara Vikberg, Esther Schoutrop, Jonas Mattsson and Isabelle Magalhaes
    Citation: Journal of Translational Medicine 2024 22:273
  8. Intratumoral injection of oncolytic viruses (OVs) shows promise in immunotherapy: ONCOS-102, a genetically engineered OV that encodes Granulocyte–Macrophage Colony-Stimulating Factor (GM-CSF) demonstrated effi...

    Authors: Neshat Saffarzadeh, Emelie Foord, Eoghan O’Leary, Rand Mahmoun, Thomas Birkballe Hansen, Victor Levitsky, Thomas Poiret and Michael Uhlin
    Citation: Journal of Translational Medicine 2024 22:250
  9. Allogeneic hematopoietic stem cell transplantation (allo-HSCT) is an effective treatment for hematological malignancies. However, viral infections, particularly EBV infection, frequently occur following allo-H...

    Authors: Xun-Hong Cao, Ze-Ying Fan, Ying-Jun Chang, Lan-Ping Xu, Xiao-Hui Zhang, Xiao-Jun Huang and Xiang-Yu Zhao
    Citation: Journal of Translational Medicine 2024 22:244
  10. Natural killer (NK) cells are unique from other immune cells in that they can rapidly kill multiple neighboring cells without the need for antigenic pre-sensitization once the cells display surface markers ass...

    Authors: Linxuan Miao, Chenglin Lu, Bin Zhang, Huili Li, Xu Zhao, Haoran Chen, Ying Liu and Xiaonan Cui
    Citation: Journal of Translational Medicine 2024 22:229
  11. The initial idea of functional tissue replacement has shifted to the concept that injected cells positively modulate myocardial healing by a non-specific immune response of the transplanted cells within the ta...

    Authors: Moritz Schweins, Ralf Gäbel, Matti Raitza, Praveen Vasudevan, Heiko Lemcke, Markus Joksch, Anna Schildt, Jens Kurth, Tobias Lindner, Felix G. Meinel, Alper Öner, Hüseyin Ince, Brigitte Vollmar, Bernd Joachim Krause, Robert David and Cajetan Immanuel Lang
    Citation: Journal of Translational Medicine 2024 22:187
  12. The clinical application of cell therapies is becoming increasingly important for the treatment of cancer, congenital immune deficiencies, and hemoglobinopathies. These therapies have been primarily manufactur...

    Authors: David F. Stroncek, Nan Zhang, Jiaqiang Ren, Rob Somerville and Anh Dinh
    Citation: Journal of Translational Medicine 2024 22:181
  13. Chimeric antigen receptor (CAR) T cell therapy has shown remarkable responses in hematological malignancies with several approved products, but not in solid tumors. Patients suffer from limited response and tu...

    Authors: Jianbin Ye, Qiaoyuan Liu, Yunxuan He, Zhenkun Song, Bao Lin, Zhiwei Hu, Juanyuan Hu, Yunshan Ning, Cheguo Cai and Yan Li
    Citation: Journal of Translational Medicine 2024 22:171
  14. MicroRNA (miRNA)-based therapies have shown great potential in myocardial repair following myocardial infarction (MI). MicroRNA-302 (miR302) has been reported to exert a protective effect on MI. However, miRNA...

    Authors: Jianjun Gu, Jia You, Hao Liang, Jiacai Zhan, Xiang Gu and Ye Zhu
    Citation: Journal of Translational Medicine 2024 22:168
  15. Skeletal system disease (SSD) is defined as a class of chronic disorders of skeletal system with poor prognosis and causes heavy economic burden. m6A, methylation at the N6 position of adenosine in RNA, is a r...

    Authors: Jianhui Liang, Qian Yi, Yang Liu, Jiachen Li, Zecheng Yang, Wei Sun and Weichao Sun
    Citation: Journal of Translational Medicine 2024 22:153
  16. Glioblastoma multiforme (GBM) is the most common malignant primary brain cancer affecting the adult population. Median overall survival for GBM patients is poor (15 months), primarily due to high rates of tumo...

    Authors: Carla Bianca Luena Victorio, Wisna Novera, Arun Ganasarajah, Joanne Ong, Melisyaa Thomas, Jonas Wu, Hilary Si Yin Toh, Alfred Xuyang Sun, Eng Eong Ooi and Ann-Marie Chacko
    Citation: Journal of Translational Medicine 2024 22:126
  17. Local skin flaps are frequently employed for wound closure to address surgical, traumatic, congenital, or oncologic defects. (1) Despite their clinical utility, skin flaps may fail due to inadequate perfusion,...

    Authors: Charlotte E. Berry, Thalia Le, Nicholas An, Michelle Griffin, Micheal Januszyk, Carter B. Kendig, Alexander Z. Fazilat, Andrew A. Churukian, Phoebe M. Pan and Derrick C. Wan
    Citation: Journal of Translational Medicine 2024 22:68
  18. Chimeric antigen receptor CAR-T cell therapies have ushered in a new era of treatment for specific blood cancers, offering unparalleled efficacy in cases of treatment resistance or relapse. However, the emerge...

    Authors: Na Zheng, Yihao Long, Zixuan Bai, Jianing Li, Hongyu Wang, Dan-Dan Song, Hong-Lin Liu, Jian-Hong Shi and Shuli Zhao
    Citation: Journal of Translational Medicine 2024 22:58
  19. Neurodegenerative diseases, including Parkinson's disease, Amyotropic Lateral Sclerosis (ALS) and Alzheimer's disease, present significant challenges for therapeutic development due to drug delivery restrictio...

    Authors: Min Hak Lee, Sukyeong Kang, Ki-Hwan Um, Seok Won Lee, Hyorin Hwang, Kyunghwa Baek and Jin Woo Choi
    Citation: Journal of Translational Medicine 2024 22:53
  20. According to the definition of the International Society for Cell and Gene Therapy (ISCT), mesenchymal stromal cells (MSCs) do not express HLA-DR. This phenotypic marker as a release criterion for clinical use...

    Authors: Zyrafete Kuçi, Natascha Piede, Kathrin Vogelsang, Lisa-Marie Pfeffermann, Sibylle Wehner, Emilia Salzmann-Manrique, Miriam Stais, Hermann Kreyenberg, Halvard Bonig, Peter Bader and Selim Kuçi
    Citation: Journal of Translational Medicine 2024 22:39
  21. Chimeric antigen receptor (CAR) T cells have shown significant activity in B-lineage malignancies. However, their efficacy in myeloid leukemia has not been successful due to unclear molecular mechanisms.

    Authors: Chuo Li, Shiyu Zuo, Lingling Shan, Huifang Huang, Haidong Cui and Xiaoming Feng
    Citation: Journal of Translational Medicine 2024 22:32
  22. Liver cancer is a major malignant tumor, which seriously threatens human health and increases the economic burden on patients. At present, gene therapy has been comprehensively studied as an excellent therapeu...

    Authors: Junhe Zhang, Yunxi Xiao, Jie Zhang, Yun Yang, Liao Zhang and Fan Liang
    Citation: Journal of Translational Medicine 2024 22:3
  23. While cartilage tissue engineering has significantly improved the speed and quality of cartilage regeneration, the underlying metabolic mechanisms are complex, making research in this area lengthy and challeng...

    Authors: Xiao-he Wang, Ning Liu, Hui Zhang, Zong-sheng Yin and Zhen-Gang Zha
    Citation: Journal of Translational Medicine 2023 21:926
  24. Osteoporosis is currently the most prevalent bone disorder worldwide and is characterized by low bone mineral density and an overall increased risk of fractures. To treat osteoporosis, a range of drugs targeti...

    Authors: Weihang Gao, Chen Liang, Ke Zhao, Mingming Hou and Yinxian Wen
    Citation: Journal of Translational Medicine 2023 21:889
  25. Defects and deficiency of AT-rich interactive domain-containing protein 1A (ARID1A) encoded by a tumor suppressor gene ARID1A have recently been suggested to get involved in angiogenesis, a crucial process in car...

    Authors: Sunisa Yoodee, Paleerath Peerapen, Sirikanya Plumworasawat, Thanyalak Malaitad and Visith Thongboonkerd
    Citation: Journal of Translational Medicine 2023 21:862
  26. In vitro or in vivo depletion of alloreactive T cells can facilitate haplo-identical hematopoietic stem cell transplantation (HSCT). Very satisfactory transplant outcomes were thus reported for TCRαβ/CD19-depl...

    Authors: E. Wiercinska, P. Quade-Lyssy, C. Hümmer, J. Beifuß, K. Akarkach, C. Poppe, V. Olevska, J. Dzionek, H. Lahnor, A. Bosio, E. Papanikolaou and Halvard Bonig
    Citation: Journal of Translational Medicine 2023 21:849
  27. Non-union formation still represents a major burden in trauma and orthopedic surgery. Moreover, aged patients are at an increased risk for bone healing failure. Parathyroid hormone (PTH) has been shown to acce...

    Authors: Maximilian M. Menger, Anne L. Tobias, David Bauer, Michelle Bleimehl, Claudia Scheuer, Michael D. Menger, Tina Histing and Matthias W. Laschke
    Citation: Journal of Translational Medicine 2023 21:844
  28. Oncolytic viruses (OVs) for cancer treatment are in a rapid stage of development, and the direct tumor lysis and activation of a comprehensive host immune response are irreplaceable advantages of cancer immuno...

    Authors: Zhen Shen, Xiyu Liu, Guixiang Fan, Jintong Na, Qiaoqiao Liu, Faquan Lin, Zhikun Zhang and Liping Zhong
    Citation: Journal of Translational Medicine 2023 21:842
  29. Patients with steroid-refractory acute graft-versus-host disease (aGvHD) not tolerating/responding to ruxolitinib (RR-aGvHD) have a dismal prognosis.

    Authors: Halvard Bonig, Mareike Verbeek, Peter Herhaus, Krischan Braitssch, Gernot Beutel, Christoph Schmid, Nadine Müller, Gesine Bug, Michaela Döring, Arend von Stackelberg, Johanna Tischer, Francis Ayuk, Gerald Wulf, Udo Holtick, Lisa-Marie Pfeffermann, Bernd Jahrsdörfer…
    Citation: Journal of Translational Medicine 2023 21:837
  30. Advancing personalized medicine in brain cancer relies on innovative strategies, with mRNA vaccines emerging as a promising avenue. While the initial use of mRNA vaccines was in oncology, their stunning succes...

    Authors: Feng Lin, Emma Z. Lin, Misa Anekoji, Thomas E. Ichim, Joyce Hu, Francesco M. Marincola, Lawrence D. Jones, Santosh Kesari and Shashaanka Ashili
    Citation: Journal of Translational Medicine 2023 21:830
  31. Extramedullary disease usually implies a dismal outcome in relapsed/refractory multiple myeloma patients, and requires novel treatment approaches. We designed a trial using Selinexor, a nuclear export protein ...

    Authors: Di Wang, Haiying Fu, Yimei Que, Haitao Ruan, Menglei Xu, Xiaolu Long, Qiuxia Yu, Chunhui Li, Zhe Li, Songbai Cai, Wei Chen, Cong Sun, Guang Hu, Shuai Wang, Donggou He, Jianming Mei…
    Citation: Journal of Translational Medicine 2023 21:812
  32. Wharton’s Jelly (WJ) Mesenchymal Stromal Cells (MSC) have emerged as an attractive allogeneic therapy for a number of indications, except for bone-related conditions requiring new tissue formation. This may be...

    Authors: Raquel Cabrera-Pérez, Alexis Ràfols-Mitjans, Ángela Roig-Molina, Silvia Beltramone, Joaquim Vives and Laura Batlle-Morera
    Citation: Journal of Translational Medicine 2023 21:802
  33. The promising potential of adeno-associated virus (AAV) gene delivery strategies to treat genetic disorders continues to grow with an additional three AAV-based therapies recently approved by the Food and Drug...

    Authors: Christina A. Pacak, Silveli Suzuki-Hatano, Fatemeh Khadir, Audrey L. Daugherty, Mughil Sriramvenugopal, Bennett J. Gosiker, Peter B. Kang and William Todd Cade
    Citation: Journal of Translational Medicine 2023 21:748
  34. Ischemia–reperfusion injury is a key complication following lung transplantation. The clinical application of ex vivo lung perfusion (EVLP) to assess donor lung function has significantly increased the utiliza...

    Authors: Jamie E. Jeon, Lei Huang, Zhiyuan Zhu, Aaron Wong, Shaf Keshavjee and Mingyao Liu
    Citation: Journal of Translational Medicine 2023 21:729
  35. Extracellular vesicles (EV) are considered a cell-free alternative to mesenchymal stromal cell (MSC) therapy. Numerous reports describe the efficacy of EV in conferring immunomodulation and promoting angiogene...

    Authors: Renata Skovronova, Eleonora Scaccia, Sandra Calcat-i-Cervera, Benedetta Bussolati, Timothy O’Brien and Karen Bieback
    Citation: Journal of Translational Medicine 2023 21:723
  36. Chimeric antigen receptor NK (CAR-NK) cell therapy is one of the most promising immunotherapies. Although it has shown a significant therapeutic effect in hematologic malignancies, few successes have been obta...

    Authors: Tingdang Liu, Ximing Dai, Yien Xu, Tian Guan, Liangli Hong, Tahir Zaib, Qi Zhou, Ke Cheng, Xiaoling Zhou, Changchun Ma and Pingnan Sun
    Citation: Journal of Translational Medicine 2023 21:710
  37. Systemic administration of oncolytic adenovirus for cancer therapy is still a challenge. Mesenchymal stem cells as cell carriers have gained increasing attention in drug delivery due to their excellent tumor t...

    Authors: Xu He, Wei Yao, Ji-Ding Zhu, Xin Jin, Xin-Yuan Liu, Kang-Jian Zhang and Shou-Liang Zhao
    Citation: Journal of Translational Medicine 2023 21:688
  38. Recent progress in cancer immunotherapy encourages the expansion of chimeric antigen receptor (CAR) T cell therapy in solid tumors including hepatocellular carcinoma (HCC). Overexpression of MET receptor tyros...

    Authors: Anna Qin, Yuan Qin, Joseph Lee, Anna Musket, Mingyao Ying, Giedre Krenciute, Francesco M. Marincola, Zhi Q. Yao, Phillip R. Musich and Qian Xie
    Citation: Journal of Translational Medicine 2023 21:682
  39. Due to environmental hypoxia on the Tibetan Plateau, local residents often exhibit a compensative increase in hemoglobin concentration to maintain the body’s oxygen supply. However, increases in hemoglobin and...

    Authors: Xiaodong Wu, Zhijuan Liu, Doudou Hao, Qin Zhao, Wanjing Li, Maodi Xie, Xia Feng, Xia Liao, Siyuan Chen, Siyu Wang, Chaohua Zhou, Wenchun Long, Yajun Zhong, Shen Li, Ye Cao, Hong Wang…
    Citation: Journal of Translational Medicine 2023 21:676
  40. Glioblastoma (GBM) is a highly aggressive primary brain tumor with a poor prognosis. This study investigates the therapeutic potential of human Vγ9Vδ2 T cells in GBM treatment. The sensitivity of different gli...

    Authors: Yi Wang, Nan Ji, Yang Zhang, Junsheng Chu, Changcun Pan, Peng Zhang, Weiwei Ma, Xueguang Zhang, Jianzhong Jeff Xi, Mingze Chen, Yonghui Zhang, Liwei Zhang and Tao Sun
    Citation: Journal of Translational Medicine 2023 21:672
  41. The chimeric antigen receptor (CAR)-T therapy has a limited therapeutic effect on solid tumors owing to the limited CAR-T cell infiltration into solid tumors and the inactivation of CAR-T cells by the immunosu...

    Authors: Yizhao Chen, Xiangling Zhu, Hanze Liu, Cunzhi Wang, Yu Chen, Huihui Wang, Yilong Fang, Xuming Wu, Yuting Xu, Chunhua Li, Xinyue Lv, Jinghua Huang, Xintong Han, Ruilin Li, Wenming Hong, Zhiying Yu…
    Citation: Journal of Translational Medicine 2023 21:654
  42. Vascularized composite allotransplantation can improve quality of life and restore functionality. However, the complex tissue composition of vascularized composite allografts (VCAs) presents unique clinical ch...

    Authors: Carolyn Ton, Sara Salehi, Sara Abasi, John R. Aggas, Renee Liu, Gerald Brandacher, Anthony Guiseppi-Elie and Warren L. Grayson
    Citation: Journal of Translational Medicine 2023 21:609
  43. Non-union formation represents a major complication in trauma and orthopedic surgery. The phosphodiesterase-5 (PDE-5) inhibitor sildenafil has been shown to exert pro-angiogenic and pro-osteogenic effects in v...

    Authors: Maximilian M. Menger, David Bauer, Michelle Bleimehl, Claudia Scheuer, Benedikt J. Braun, Steven C. Herath, Mika F. Rollmann, Michael D. Menger, Matthias W. Laschke and Tina Histing
    Citation: Journal of Translational Medicine 2023 21:607
  44. Cell-based strategies focusing on replacement or protection of dopaminergic neurons have been considered as a potential approach to treat Parkinson’s disease (PD) for decades. However, despite promising precli...

    Authors: Fang Wang, Zhengwu Sun, Daoyong Peng, Shikha Gianchandani, Weidong Le, Johannes Boltze and Shen Li
    Citation: Journal of Translational Medicine 2023 21:601
  45. Ovarian cancer (OC) is a highly aggressive gynecological malignancy prevalent worldwide. Most OC cases are typically diagnosed at advanced stages, which has led to a 5-year overall survival rate of less than 3...

    Authors: Haihong Zhao, Lina Wu, Jiemin Dai, Ke Sun, Zhenguo Zi, Junhua Guan and Liwen Zhang
    Citation: Journal of Translational Medicine 2023 21:596
  46. Cyclooxygenase (COX)-2 is a rate-limiting enzyme in the biosynthesis of prostanoids, which is mostly inducible by inflammatory cytokines. The participation of COX-2 in the maturation of megakaryocytes has been...

    Authors: Xibing Zhuang, Pengcheng Xu, Yang Ou, Xia Shao, Ying Li, Yanna Ma, Shanshan Qin, Fanli Hua, Yanxia Zhan, Lili Ji, Tiankui Qiao, Hao Chen and Yunfeng Cheng
    Citation: Journal of Translational Medicine 2023 21:540
  47. Glioblastoma (GBM) is recognized as among the most aggressive forms of brain tumor. Patients typically present with a five-year survival rate of less than 6% with traditional surgery and chemoradiotherapy, whi...

    Authors: Shijie Wang, Wenwen Wei, Yuncang Yuan, Bin Sun, Dong Yang, Nan Liu and Xudong Zhao
    Citation: Journal of Translational Medicine 2023 21:493

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