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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 

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  1. 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:

  2. 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:

  3. 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:

  4. 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:

  5. 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:

  6. 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:

  7. 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:

  8. 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:

  9. A novel approach to treating heart failures was developed with the introduction of iPSC technology. Knowledge in regenerative medicine, developmental biology, and the identification of illnesses at the cellula...

    Authors: Seyedeh Parya Farboud, Ezzatollah Fathi, Behnaz Valipour and Raheleh Farahzadi
    Citation: Journal of Translational Medicine 2024 22:783
    Content type: Review Published on:

  10. The skin epidermis is continually influenced by a myriad of internal and external elements. At its basal layer reside epidermal stem cells, which fuels epidermal renovation and hair regeneration with powerful ...

    Authors: Xin Tang, Jiaqi Wang, Jiaoling Chen, Wanting Liu, Pei Qiao, Huiyi Quan, Zhiguo Li, Erle Dang, Gang Wang and Shuai Shao
    Citation: Journal of Translational Medicine 2024 22:779
    Content type: Review Published on:

  11. Biobanks have become an integral part of health and bioscience research. However, the ultra-low temperature (ULT) storage methods that biobanks employ [ULT freezers and liquid nitrogen (LN2)] are associated wi...

    Authors: Matthew Graham, Gabrielle Samuel and Martin Farley
    Citation: Journal of Translational Medicine 2024 22:747
    Content type: Review Published on:

  12. Asthma poses a major public health burden. While existing asthma drugs manage symptoms for many, some patients remain resistant. The lack of a cure, especially for severe asthma, compels exploration of novel t...

    Authors: Mohadeseh Mohammad Taheri, Fatemeh Javan, Mohadeseh Poudineh and Seyed Shamseddin Athari
    Citation: Journal of Translational Medicine 2024 22:736
    Content type: Review Published on:

  14. The immunogenicity of the antigen-recognition domains of chimeric antigen receptor (CAR)-T cells leads to immune responses that may compromise the antitumor effects of the adoptively transferred T cells. Herei...

    Authors: Pooria Safarzadeh Kozani, Pouya Safarzadeh Kozani and Fatemeh Rahbarizadeh
    Citation: Journal of Translational Medicine 2024 22:679
    Content type: Research Published on:

  15. Pancreatic cancer is one of the most lethal malignancies and the lack of treatment options makes it more deadly. Chimeric Antigen Receptor T-cell (CAR-T) immunotherapy has revolutionized cancer treatment and m...

    Authors: Tian Deng, Yingzhi Deng, Shih-Ting Tsao, Qinghui Xiong, Yue Yao, Cuicui Liu, Ming yuan Gu, Fei Huang and Haiying Wang
    Citation: Journal of Translational Medicine 2024 22:633
    Content type: Research Published on:

  16. Mesenchymal stem and stromal cells (MSCs) hold potential to treat a broad range of clinical indications, but clinical translation has been limited to date due in part to challenges with batch-to-batch reproduc...

    Authors: Brandon Krupczak, Camille Farruggio and Krystyn J. Van Vliet
    Citation: Journal of Translational Medicine 2024 22:614
    Content type: Research Published on:

  17. CD19-targeted chimeric antigen receptor T (CAR-T) cell therapy stands out as a revolutionary intervention, exhibiting remarkable remission rates in patients with refractory/relapsed (R/R) B-cell malignancies. ...

    Authors: Lian-Fang Pu, Hui-Min Zheng, Xiang-Jiang Feng, Alice Charwudzi, Xue Liang, Lin-Hui Hu, Yang-Yang Ding, Ze-Lin Liu, Ya Liao and Shu-Dao Xiong
    Citation: Journal of Translational Medicine 2024 22:613
    Content type: Research Published on:

  18. The application of graphene-based nanocomposites for therapeutic and diagnostic reasons has advanced considerably in recent years due to advancements in the synthesis and design of graphene-based nanocomposite...

    Authors: Mahnaz Asadi, Seyed Hosein Ghorbani, Leila Mahdavian and Mohammad Aghamohammadi
    Citation: Journal of Translational Medicine 2024 22:611
    Content type: Review Published on:

  19. Patients with hemorrhagic shock and trauma (HS/T) are vulnerable to the endotheliopathy of trauma (EOT), characterized by vascular barrier dysfunction, inflammation, and coagulopathy. Cellular therapies such a...

    Authors: Mark Barry, Alpa Trivedi, Byron Miyazawa, Lindsay R. Vivona, David Shimmin, Praneeti Pathipati, Callie Keane, Joseph Cuschieri and Shibani Pati
    Citation: Journal of Translational Medicine 2024 22:588
    Content type: Research Published on:

  20. The coronavirus disease 2019 (COVID-19) has become a serious public health issue. In COVID-19 patients, the elevated levels of inflammatory cytokines lead to the manifestation of COVID-19 symptoms, such as lun...

    Authors: Wenming Lu, Longxiang Yan, Xingkun Tang, Xuesong Wang, Jing Du, Zhengwei Zou, Lincai Li, Junsong Ye and Lin Zhou
    Citation: Journal of Translational Medicine 2024 22:550
    Content type: Review Published on:

  21. Despite significant advancements in treatment strategies, multiple myeloma remains incurable. Additionally, there is a distinct lack of reliable biomarkers that can guide initial treatment decisions and help d...

    Authors: Sigrid Ravn Berg, Aida Dikic, Animesh Sharma, Lars Hagen, Cathrine Broberg Vågbø, Alexey Zatula, Kristine Misund, Anders Waage and Geir Slupphaug
    Citation: Journal of Translational Medicine 2024 22:548
    Content type: Research Published on:

  22. Dendritic cells (DCs) have been intensively studied in correlation to tumor immunology and for the development DC-based cancer vaccines. Here, we present the significance of the temporal aspect of DC maturatio...

    Authors: Primož Poženel, Kaja Zajc and Urban Švajger
    Citation: Journal of Translational Medicine 2024 22:541
    Content type: Research Published on:

  23. Macrophages are involved in tissue homeostasis, angiogenesis and immunomodulation. Proangiogenic and anti-inflammatory macrophages (regulatory macrophages, Mreg) can be differentiated in-vitro from CD14+ monocyte...

    Authors: Karina Zitta, Lars Hummitzsch, Frank Lichte, Fred Fändrich, Markus Steinfath, Christine Eimer, Sebastian Kapahnke, Matthias Buerger, Katharina Hess, Melanie Rusch, Rene Rusch, Rouven Berndt and Martin Albrecht
    Citation: Journal of Translational Medicine 2024 22:534
    Content type: Research Published on:

  24. Neutrophils are granulocytes with essential antimicrobial effector functions and short lifespans. During infection or sterile inflammation, emergency granulopoiesis leads to release of immature neutrophils fro...

    Authors: Claire A. Naveh, Kiran Roberts, Przemysław Zakrzewski, Christopher M. Rice, Fernando M. Ponce-Garcia, Kathryn Fleming, Megan Thompson, Nawamin Panyapiean, Huan Jiang, Stephanie Diezmann, Pedro L. Moura, Ashley M. Toye and Borko Amulic
    Citation: Journal of Translational Medicine 2024 22:526
    Content type: Research Published on:

    The Correction to this article has been published in Journal of Translational Medicine 2024 22:899

  25. Autologous bone grafting is the standard treatment for the surgical management of atrophic nonunion of long bones. Other solutions, such as bone marrow mesenchymal stem cells (BM-MSC) combined with phospho-cal...

    Authors: Lluís Orozco Delclós, Robert Soler Rich, Rafael Arriaza Loureda, Alonso Moreno García and Enrique Gómez Barrena
    Citation: Journal of Translational Medicine 2024 22:493
    Content type: Research Published on:

  26. Cellular immunotherapy, represented by the chimeric antigen receptor T cell (CAR-T), has exhibited high response rates, durable remission, and safety in vitro and in clinical trials. Unfortunately, anti-CD19 C...

    Authors: Hongzhe Li, Yuwen Wang, Rongrong Liu, Xiaoxiao Li, Ping Zhang, Ping Chen, Ning Zhao, Bing Li, Jie Wang and Yongmin Tang
    Citation: Journal of Translational Medicine 2024 22:482
    Content type: Research Published on:

  27. Inflammation plays a critical role in conditions such as acute liver failure, acute-on-chronic liver failure, and ischemia–reperfusion-induced liver injury. Various pathogenic pathways contribute to liver infl...

    Authors: Alexandre Sitbon, Pierre-Romain Delmotte, Valéria Pistorio, Sébastien Halter, Jérémy Gallet, Jérémie Gautheron and Antoine Monsel
    Citation: Journal of Translational Medicine 2024 22:480
    Content type: Review Published on:

  28. Mesenchymal stem cells (MSCs) have garnered significant interest for their tumor-tropic property, making them potential therapeutic delivery vehicles for cancer treatment. We have previously shown the signific...

    Authors: Yoon Khei Ho, Jun Yung Woo, Kin Man Loke, Lih-Wen Deng and Heng-Phon Too
    Citation: Journal of Translational Medicine 2024 22:463
    Content type: Research Published on:

  30. One of the most challenging aspects of developing advanced cell therapy products (CTPs) is defining the mechanism of action (MOA), potency and efficacy of the product. This perspective examines these concepts ...

    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 2024 22:416
    Content type: Review Published on:

  31. Adoptive cell therapy has revolutionized cancer treatment, especially for hematologic malignancies. T cells are the most extensively utilized cells in adoptive cell therapy. Currently, tumor-infiltrating lymph...

    Authors: Yinqi Li, Yeteng Zheng, Taiqing Liu, Chuanyun Liao, Guobo Shen and Zhiyao He
    Citation: Journal of Translational Medicine 2024 22:413
    Content type: Review Published on:

  32. The immune system in humans is a defense department against both exogenous and endogenous hazards, where CD8+ T cells play a crucial role in opposing pathological threats. Various immunotherapies based on CD8+ T ...

    Authors: Yanxia Chen, Dingning Yu, Hui Qian, Yinghong Shi and Zhimin Tao
    Citation: Journal of Translational Medicine 2024 22:394
    Content type: Review Published on:

  33. Chimeric antigen receptor (CAR) T-cells have demonstrated significant efficacy in targeting hematological malignancies, and their use continues to expand. Despite substantial efforts spent on the optimization ...

    Authors: Michaela Prochazkova, Alexandra Dreyzin, Lipei Shao, Pam Garces, Yihua Cai, Rongye Shi, Alejandra Pelayo, Yong Soo Kim, Victoria Pham, Sue Ellen Frodigh, Shannon Fenton, Catherine Karangwa, Yan Su, Kathryn Martin, Nan Zhang, Steven L. Highfill…
    Citation: Journal of Translational Medicine 2024 22:384
    Content type: Research Published on:

  34. In this study, we investigated CD70 as a promising target for renal cell carcinoma (RCC) therapy and developed a potent chimeric antigen receptor T (CAR-T) cells for potential clinical testing. CD70, found to ...

    Authors: Qinghui Xiong, Haiying Wang, Qiushuang Shen, Yan Wang, Xiujie Yuan, Guangyao Lin and Pengfei Jiang
    Citation: Journal of Translational Medicine 2024 22:368
    Content type: Research Published on:

  35. Ovarian cancer (OC) is characterized by its rapid growth and spread which, accompanied by a low 5-year survival rate, necessitates the development of improved treatments. In ovarian cancer, the selective overe...

    Authors: Jing Guo, Xiaozhu Zeng, Yongjie Zhu, Dong Yang and Xudong Zhao
    Citation: Journal of Translational Medicine 2024 22:367
    Content type: Research Published on:

  36. 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
    Content type: Research Published on:

  38. 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
    Content type: Review Published on:

  39. 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
    Content type: Research Published on:

  40. 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
    Content type: Research Published on:

  41. 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
    Content type: Research Published on:

  42. 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
    Content type: Research Published on:

  43. 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
    Content type: Research Published on:

  44. 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
    Content type: Research Published on:

  45. 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
    Content type: Research Published on:

  46. 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
    Content type: Review Published on:

  47. 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
    Content type: Research Published on:

  48. 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
    Content type: Commentary Published on:

  49. 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
    Content type: Research Published on:

  50. 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
    Content type: Research Published on:
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