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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. Extracellular vesicles (ECV) and bone extracellular matrix (ECM) have beneficial effects on the treatment of some pathological conditions. The purpose of this study was to find the synergic effects of decellul...

    Authors: Asrin Emami, Tahereh Talaei-Khozani, Saeid Tavanafar, Nehleh Zareifard, Negar Azarpira and Zahra Vojdani
    Citation: Journal of Translational Medicine 2020 18:361
    Content type: Research Published on:

  2. Mesenchymal stromal cells are a safe and promising option to treat knee osteoarthritis as previously demonstrated in different clinical trials. However, their efficacy, optimal dose and addition of adjuvants m...

    Authors: José María Lamo-Espinosa, Juan F. Blanco, Mikel Sánchez, Victoria Moreno, Froilán Granero-Moltó, Fermín Sánchez-Guijo, Íñigo Crespo-Cullel, Gonzalo Mora, Diego Delgado San Vicente, Orlando Pompei-Fernández, Jesús Dámaso Aquerreta, Jorge María Núñez-Córdoba, María Vitoria Sola, Andrés Valentí-Azcárate, Enrique J. Andreu, María del Consuelo del Cañizo…
    Citation: Journal of Translational Medicine 2020 18:356
    Content type: Research Published on:

  3. Foetal bovine serum (FBS), is the most commonly used culture medium additive for in vitro cultures, despite its undefined composition, its potential immunogenicity and possible prion/zoonotic transmission. For...

    Authors: M. Guiotto, W. Raffoul, A. M. Hart, M. O. Riehle and P. G. di Summa
    Citation: Journal of Translational Medicine 2020 18:351
    Content type: Review Published on:

  5. Deceased organ donors represent an untapped source of therapeutic bone marrow (BM) that can be recovered in 3–5 times the volume of that obtained from living donors, tested for quality, cryopreserved, and bank...

    Authors: Erik J. Woods, Aubrey M. Sherry, John R. Woods, James W. Hardin, Michael LaFontaine, Gerald Brandacher and Brian H. Johnstone
    Citation: Journal of Translational Medicine 2020 18:300
    Content type: Research Published on:

  6. Treatment of hepatocellular carcinoma (HCC) using antibody-based targeted therapies, such as antibody conjugates and chimeric antigen receptor T (CAR-T) cell therapy, shows potent antitumor efficacy. Glypican-...

    Authors: Xiaoyu Liu, Fang Gao, Longwei Jiang, Meng Jia, Lei Ao, Ming Lu, Liming Gou, Mitchell Ho, Shaochang Jia, Fei Chen and Wei Gao
    Citation: Journal of Translational Medicine 2020 18:295
    Content type: Research Published on:

  7. Although a large body of information exists relating to cellular therapies, much of this information is either anecdotal or has been obtained from relatively small clinical trials, so that the level of evidenc...

    Authors: Amanda Lindeman, Carl J. Pepine and Keith L. March
    Citation: Journal of Translational Medicine 2020 18:285
    Content type: Research Published on:

  8. Mesenchymal stem cells (MSCs) have been shown to alleviate acute lung injury (ALI) and induce the production of regulatory dendritic cells (DCregs), but the potential link between these two cell types remains ...

    Authors: Zhonghua Lu, Shanshan Meng, Wei Chang, Shanwen Fan, Jianfeng Xie, Fengmei Guo, Yi Yang, Haibo Qiu and Ling Liu
    Citation: Journal of Translational Medicine 2020 18:241
    Content type: Research Published on:

  9. The in vitro production of mature human red blood cells (RBCs) from induced pluripotent stem cells (iPSCs) has been the focus of research to meet the high demand for blood transfusions. However, limitations li...

    Authors: Yu Jin Park, Su-Hee Jeon, Hyun-Kyung Kim, Eun Jung Suh, Seung Jun Choi, Sinyoung Kim and Hyun Ok Kim
    Citation: Journal of Translational Medicine 2020 18:236
    Content type: Research Published on:

  10. Typically minor ABO incompatible platelet products are transfused without any incident, yet serious hemolytic transfusion reactions occur. To mitigate these events, ABO ‘low titer’ products are used for minor ...

    Authors: Priyanka Pandey, Waseem Q. Anani, Tina Pugh, Jerome L. Gottschall and Gregory A. Denomme
    Citation: Journal of Translational Medicine 2020 18:216
    Content type: Research Published on:

  11. In late 2019, a novel coronavirus (SARS-CoV-2) emerged in Wuhan, capital city of Hubei province in China. Cases of SARS-CoV-2 infection quickly grew by several thousand per day. Less than 100 days later, the W...

    Authors: Christopher J. Rogers, Robert J. Harman, Bruce A. Bunnell, Martin A. Schreiber, Charlie Xiang, Fu-Sheng Wang, Antonio F. Santidrian and Boris R. Minev
    Citation: Journal of Translational Medicine 2020 18:203
    Content type: Review Published on:

  12. Glioblastoma stem-like cells (GSCs) are hypothesized to contribute to self-renewal and therapeutic resistance in glioblastoma multiforme (GBM) tumors. Constituting only a small percentage of cancer cells, GSCs...

    Authors: Xu Wang, Wenjuan Zhou, Xian Li, Jun Ren, Guangyu Ji, Jingyi Du, Wenyu Tian, Qian Liu and Aijun Hao
    Citation: Journal of Translational Medicine 2020 18:200
    Content type: Research Published on:

  13. We present the rationale for a novel allo-priming approach to serve the elderly as a universal anti-virus vaccine, as well serving to remodel the aging immune system in order to reverse immunosenescence and in...

    Authors: Michael Har-Noy and Reuven Or
    Citation: Journal of Translational Medicine 2020 18:196
    Content type: Review Published on:

  14. Genetically engineered T cells have become an important therapy for B-cell malignancies. Measuring the efficiency of vector integration into the T cell genome is important for assessing the potency and safety ...

    Authors: Alex Lu, Hui Liu, Rongye Shi, Yihua Cai, Jinxia Ma, Lipei Shao, Victor Rong, Nikolaos Gkitsas, Hong Lei, Steven L. Highfill, Sandhya Panch, David F. Stroncek and Ping Jin
    Citation: Journal of Translational Medicine 2020 18:191
    Content type: Research Published on:

  15. Progressive multifocal leukoencephalopathy is a demyelinating CNS disorder. Reactivation of John Cunningham virus leads to oligodendrocyte infection with lysis and consequent axonal loss due to demyelination. ...

    Authors: M. J. Steinhardt, E. Wiercinska, M. Pham, G. U. Grigoleit, A. Mazzoni, M. Da-Via, X. Zhou, K. Meckel, K. Nickel, J. Duell, F. C. Krummenast, S. Kraus, C. Hopkinson, B. Weissbrich, W. Müllges, G. Stoll…
    Citation: Journal of Translational Medicine 2020 18:177
    Content type: Methodology Published on:

  16. Kidney ischemia reperfusion injury (IRI) is a common cause of acute kidney injury and an unavoidable consequence of kidney transplantation and still lacks specific therapeutics. Recently, mesenchymal stem cell...

    Authors: Shuo Wang, Songjie Cai, Weitao Zhang, Xigao Liu, Yan Li, Chao Zhang, Yigang Zeng, Ming Xu, Ruiming Rong, Tianshu Yang, Benkang Shi, Anil Chandraker, Cheng Yang and Tongyu Zhu
    Citation: Journal of Translational Medicine 2020 18:175
    Content type: Research Published on:

  17. Adoptive transfer of virus-specific T cells (VSTs) represents a prophylactic and curative approach for opportunistic viral infections and reactivations after transplantation. However, inadequate frequencies of...

    Authors: Nele Carolin Heinemann, Sabine Tischer-Zimmermann, Torge Christian Wittke, Julian Eigendorf, Arno Kerling, Theodor Framke, Anette Melk, Hans-Gert Heuft, Rainer Blasczyk, Britta Maecker-Kolhoff and Britta Eiz-Vesper
    Citation: Journal of Translational Medicine 2020 18:148
    Content type: Research Published on:

  18. The skin is the largest organ of the human body. Upon injury, the skin triggers a sequence of signaling pathways that induce epithelial proliferation, migration, and ultimately, the re-establishment of the epi...

    Authors: Carlos H. V. Nascimento-Filho, Ericka J. D. Silveira, Eny M. Goloni-Bertollo, Lelia Batista de Souza, Cristiane H. Squarize and Rogerio M. Castilho
    Citation: Journal of Translational Medicine 2020 18:138
    Content type: Research Published on:

  19. Cell proliferation and death are key components of wound healing and tissue repair. Telocytes (TCs) represent a newly discovered cell type that can protect tissue from acute injury via cell–cell communication ...

    Authors: Lu Wang, Dongli Song, Chuanyuan Wei, Cheng Chen, Yanwen Yang, Xinyi Deng and Jianying Gu
    Citation: Journal of Translational Medicine 2020 18:60
    Content type: Research Published on:

  20. Tissue-engineered skin (TES), as an analogue of native skin, is promising for wound repair and regeneration. However, a major drawback of TES products is a lack of skin appendages and nerves to enhance skin he...

    Authors: Tingting Weng, Pan Wu, Wei Zhang, Yurong Zheng, Qiong Li, Ronghua Jin, Haojiao Chen, Chuangang You, Songxue Guo, Chunmao Han and Xingang Wang
    Citation: Journal of Translational Medicine 2020 18:53
    Content type: Review Published on:

  21. Mesenchymal stromal cells (MSCs) have attracted intense interest due to their powerful intrinsic properties of self-regeneration, immunomodulation and multi-potency, as well as being readily available and easy...

    Authors: Dickson Kofi Wiredu Ocansey, Bing Pei, Yongmin Yan, Hui Qian, Xu Zhang, Wenrong Xu and Fei Mao
    Citation: Journal of Translational Medicine 2020 18:42
    Content type: Review Published on:

  22. Bone marrow mononuclear cells have been successfully utilized for numerous regenerative purposes. In the current study, patients suffering from erectile dysfunction (ED) unresponsive to phosphodiesterase 5 inh...

    Authors: Mark Bieri, Elias Said, Gabrielle Antonini, Donald Dickerson, Jorge Tuma, Courtney E. Bartlett, Amit N. Patel and Alexander Gershman
    Citation: Journal of Translational Medicine 2020 18:24
    Content type: Research Published on:

  23. Prostate cancer is one of the most common adult malignancies in men, and nearly all patients with metastatic prostate cancer can develop and receive resistance to primary androgen deprivation therapy (ADT), a ...

    Authors: Dawei Wang, Yuan Shao, Xiang Zhang, Guoliang Lu and Boke Liu
    Citation: Journal of Translational Medicine 2020 18:23
    Content type: Research Published on:

  24. Malignant behavior and radioresistance, which severely limits the efficacy of radiation therapy (RT) in nasopharyngeal carcinoma (NPC), are associated with tumor progression and poor prognosis. Mesenchymal ste...

    Authors: Fang-Zhu Wan, Kai-Hua Chen, Yong-Chu Sun, Xi-Chan Chen, Ren-Ba Liang, Li Chen and Xiao-Dong Zhu
    Citation: Journal of Translational Medicine 2020 18:12
    Content type: Research Published on:

  25. A worldwide lack of donor corneas demands the bioengineered corneas be developed as an alternative. The primary objective of the current study was to evaluate the efficacy of acellular porcine corneal stroma (...

    Authors: Saiqun Li, Meng Li, Li Gu, Lulu Peng, Yuqing Deng, Jing Zhong, Bowen Wang, Qian Wang, Yichen Xiao and Jin Yuan
    Citation: Journal of Translational Medicine 2019 17:434
    Content type: Research Published on:

  26. Innovative human stromal cell therapeutics require xeno-free culture conditions. Various formulations of human platelet lysate (HPL) are efficient alternatives for fetal bovine serum (FBS). However, a consiste...

    Authors: Sandra Laner-Plamberger, Michaela Oeller, Cornelia Mrazek, Arnulf Hartl, Alina Sonderegger, Eva Rohde, Dirk Strunk and Katharina Schallmoser
    Citation: Journal of Translational Medicine 2019 17:432
    Content type: Research Published on:

  27. Telocytes (TCs) have the capacity of cell–cell communication with adjacent cells within the tissue, contributing to tissue repair and recovery from injury. The present study aims at investigating the molecular...

    Authors: Dongli Song, Li Tang, Jianan Huang, Lu Wang, Tao Zeng and Xiangdong Wang
    Citation: Journal of Translational Medicine 2019 17:431
    Content type: Research Published on:

  28. Delayed bone healing, especially in long bones poses one of the biggest problems in orthopeadic and reconstructive surgery and causes tremendous costs every year. There is a need for exploring the causes in or...

    Authors: Johannes M. Wagner, Sonja V. Schmidt, Mehran Dadras, Julika Huber, Christoph Wallner, Stephanie Dittfeld, Mustafa Becerikli, Henriette Jaurich, Felix Reinkemeier, Marius Drysch, Marcus Lehnhardt and Björn Behr
    Citation: Journal of Translational Medicine 2019 17:416
    Content type: Research Published on:

  29. The liver is supplied by a dual blood supply, including the portal venous system and the hepatic arterial system; thus, the liver organ is exposed to multiple gut microbial products, metabolic products, and to...

    Authors: Chenxia Hu and Lanjuan Li
    Citation: Journal of Translational Medicine 2019 17:412
    Content type: Review Published on:

  30. Mesenchymal stem cells (MSCs) represent an invaluable asset for the field of cell therapy. Human Bone marrow-derived MSCs (hBM-MSCs) are one of the most commonly used cell types in clinical trials. They are cu...

    Authors: Soukaina Bahsoun, Karen Coopman and Elizabeth C. Akam
    Citation: Journal of Translational Medicine 2019 17:397
    Content type: Review Published on:

  31. The aim of this systematic review study is to summarize the current knowledge of ovarian tissue transplantation and provide insight on ovarian function, fertility and reproductive outcome following ovarian tis...

    Authors: Sepideh Sheshpari, Mahnaz Shahnazi, Halimeh Mobarak, Shahin Ahmadian, Alberto Miranda Bedate, Ziba Nariman-Saleh-Fam, Mohammad Nouri, Reza Rahbarghazi and Mahdi Mahdipour
    Citation: Journal of Translational Medicine 2019 17:396
    Content type: Review Published on:

  33. Liver, as a vital organ, is responsible for a wide range of biological functions to maintain homeostasis and any type of damages to hepatic tissue contributes to disease progression and death. Viral infection,...

    Authors: Vahid Hosseini, Nazila Fathi Maroufi, Sepideh Saghati, Nahideh Asadi, Masoud Darabi, Saeed Nazari Soltan Ahmad, Hosseini Hosseinkhani and Reza Rahbarghazi
    Citation: Journal of Translational Medicine 2019 17:383
    Content type: Review Published on:

  34. When bone marrow is repeatedly filtered through porous material, the mesenchymal stem cells (MSCs) in the bone marrow can adhere to the outer and inner walls of the carrier material to become enriched locally,...

    Authors: Wenxiang Chu, Yifu Zhuang, Yaokai Gan, Xin Wang, Tingting Tang and Kerong Dai
    Citation: Journal of Translational Medicine 2019 17:377
    Content type: Research Published on:

  36. The colorectum includes ascending colon, transverse colon, descending colon, sigmoid colon, and rectum. Different sites of colorectal cancer (CRC) are different in many aspects, including clinical symptoms, bi...

    Authors: Yang Xi, Pan Yuefen, Wu Wei, Qi Quan, Zhuang Jing, Xu Jiamin and Han Shuwen
    Citation: Journal of Translational Medicine 2019 17:353
    Content type: Research Published on:

  37. Liver cancer is the second leading causes of cancer-related death globally. Pyrroline-5-carboxylate reductase 1 (PYCR1) plays a critical role in metabolic profiles of tumors. Therefore, it is necessary to expl...

    Authors: Juhua Zhuang, Yanan Song, Ying Ye, Saifei He, Xing Ma, Miao Zhang, Jing Ni, Jiening Wang and Wei Xia
    Citation: Journal of Translational Medicine 2019 17:343
    Content type: Research Published on:

  38. The P38 mitogen-activated protein kinase (MAPK) pathway plays an essential role in CVB3-induced diseases. We previously demonstrated microRNA-21 has potential inhibitory effect on the MAP2K3 which locates upst...

    Authors: Feng He, Zonghui Xiao, Hailan Yao, Sen Li, Miao Feng, Wei Wang, Zhewei Liu, Zhuo Liu and Jianxin Wu
    Citation: Journal of Translational Medicine 2019 17:335
    Content type: Research Published on:

  39. Mesenchymal stem cells (MSCs) are attractive cell-therapy candidates. Despite their popularity and promise, there is no uniform method of preparation of MSCs. Typically, cells are cryopreserved in liquid nitro...

    Authors: Ben Antebi, Amber M. Asher, Luis A. Rodriguez II, Robbie K. Moore, Arezoo Mohammadipoor and Leopoldo C. Cancio
    Citation: Journal of Translational Medicine 2019 17:297
    Content type: Research Published on:

  40. Glutathione S-transferase A3 (GSTA3) is known as an antioxidative protease, however, the crucial role of GSTA3 in liver fibrosis remains unclear. As a recently we developed water-soluble pyridone agent with antif...

    Authors: Haihua Chen, Qixin Gan, Congying Yang, Xiongqun Peng, Jiao Qin, Sisi Qiu, Yanzhi Jiang, Sha Tu, Ying He, Shenglan Li, Huixiang Yang, Lijian Tao and Yu Peng
    Citation: Journal of Translational Medicine 2019 17:280
    Content type: Research Published on:

    The Correction to this article has been published in Journal of Translational Medicine 2020 18:182

  41. Deep venous thrombosis (DVT) of lower extremities is a common thrombotic disease, occurring either in isolation or as a complication of other diseases or procedures. MiR-21 is one of important microRNAs which ...

    Authors: Xiaolong Du, Lei Hong, Lili Sun, Hongfei Sang, Aiming Qian, Wendong Li, Hao Zhuang, Huoqi Liang, Dandan Song, Chenglong Li, Wenbin Wang and Xiaoqiang Li
    Citation: Journal of Translational Medicine 2019 17:270
    Content type: Research Published on:

  42. Mesenchymal stromal cells (MSC) demonstrate innate and regulatory immunologic functions and have been widely explored for cell therapy applications. Mechanisms by which MSCs achieve therapeutic effects are the...

    Authors: Alexandra Burr and Biju Parekkadan
    Citation: Journal of Translational Medicine 2019 17:263
    Content type: Research Published on:

  43. Cell therapy has been proposed for patients with critical limb ischemia (CLI). Autologous bone marrow derived cells (BMCs) have been mostly used, mesenchymal stem cells (MSCs) being an alternative. The aim of ...

    Authors: Rida Al-Rifai, Philippe Nguyen, Nicole Bouland, Christine Terryn, Lukshe Kanagaratnam, Gaël Poitevin, Caroline François, Catherine Boisson-Vidal, Marie-Antoinette Sevestre and Claire Tournois
    Citation: Journal of Translational Medicine 2019 17:261
    Content type: Research Published on:

  44. The rapid evolution of cell-based immunotherapies such as chimeric antigen receptor T-cells for treatment of hematological cancers has precipitated the need for a platform to expand these cells ex vivo in a sa...

    Authors: Claire Coeshott, Boah Vang, Mark Jones and Brian Nankervis
    Citation: Journal of Translational Medicine 2019 17:258
    Content type: Research Published on:

  45. Diabetic cardiomyopathy (DCM) is a common complication of diabetes and is characterized by chronic myocardial inflammation. Mesenchymal stem cell (MSC) infusions have recently been suggested to alleviate myoca...

    Authors: Liyuan Jin, Zihui Deng, Jinying Zhang, Chen Yang, Jiejie Liu, Weidong Han, Ping Ye, Yiling Si and Guanghui Chen
    Citation: Journal of Translational Medicine 2019 17:251
    Content type: Research Published on:

  46. Here, we isolated, expanded and functionally characterized regulatory T cells (Tregs) from patients with end stage kidney and liver disease, waiting for kidney/liver transplantation (KT/LT), with the aim to es...

    Authors: Francesca Ulbar, Tiziana Montemurro, Tatiana Jofra, Miriam Capri, Giorgia Comai, Valentina Bertuzzo, Cristiana Lavazza, Alessandra Mandelli, Mariele Viganò, Silvia Budelli, Maria Giulia Bacalini, Chiara Pirazzini, Paolo Garagnani, Valeria Giudice, Daria Sollazzo, Antonio Curti…
    Citation: Journal of Translational Medicine 2019 17:250
    Content type: Research Published on:

  47. The relationship between the tissue injury healing response and development of heterotopic ossification (HO) is poorly understood. Here we compare a rat blast model and human traumatized muscle from a blast in...

    Authors: Jaira F. de Vasconcellos, Sonia Zicari, Stephen D. Fernicola, Daniel W. Griffin, Youngmi Ji, Emily H. Shin, Patrick Jones, Gregory T. Christopherson, Husain Bharmal, Carl Cirino, Thao Nguyen, Astor Robertson, Vincent D. Pellegrini Jr. and Leon J. Nesti
    Citation: Journal of Translational Medicine 2019 17:248
    Content type: Research Published on:

  48. The molecular pathways that drive bone marrow myeloid progenitors (BMMP) development are very well understood and include a tight controlled multi-stage gene hierarch. Monocytes are versatile cells that displa...

    Authors: Mariana Cabanel, Thayse Pinheiro da Costa, Marcia Cury EL-Cheikh and Katia Carneiro
    Citation: Journal of Translational Medicine 2019 17:247
    Content type: Research Published on:

  49. Cell-based therapies have the potential to become treatment options for many diseases, but efficient scale-out of these therapies has proven to be a major hurdle. Bioreactors can be used to overcome this hurdl...

    Authors: Ruud Das, Rens Roosloot, Melissa van Pel, Koen Schepers, Marijn Driessen, Willem E. Fibbe, Joost Dick de Bruijn and Helene Roelofs
    Citation: Journal of Translational Medicine 2019 17:241
    Content type: Research Published on:

  50. The use of “off-the-shelf” cellular therapy products derived from healthy donors addresses many of the challenges associated with customized cell products. However, the potential of allogeneic cell products to...

    Authors: David H. Quach, Luis Becerra-Dominguez, Rayne H. Rouce and Cliona M. Rooney
    Citation: Journal of Translational Medicine 2019 17:240
    Content type: Research Published on:
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