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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. Chronic low back pain due to disc degeneration represents a major social and economic burden worldwide. The current standard of care is limited to symptomatic relief and no current approved therapy promotes di...

    Authors: Christian Elabd, Christopher J. Centeno, John R. Schultz, Gregory Lutz, Thomas Ichim and Francisco J. Silva
    Citation: Journal of Translational Medicine 2016 14:253
  2. Non-healing wounds are a major global health concern and account for the majority of non-traumatic limb amputations worldwide. However, compared to standard care practices, few advanced therapeutics effectivel...

    Authors: Anthony R. Sheets, Conner J. Massey, Stephen M. Cronk, Mark D. Iafrati and Ira M. Herman
    Citation: Journal of Translational Medicine 2016 14:197
  3. Stromal vascular fraction (SVF) can easily be obtained from a mini-lipoaspirate procedure of fat tissue. The SVF contains a mixture of cells including ADSCs and growth factors and has been depleted of the adip...

    Authors: K. Comella, J. Parcero, H. Bansal, J. Perez, J. Lopez, A. Agrawal and T. Ichim
    Citation: Journal of Translational Medicine 2016 14:158
  4. Mesenchymal stromal cells (MSC) are largely investigated in clinical trials aiming to control inappropriate immune reactions (GVHD, Crohn’s disease, solid organ transplantation). As the percentage of MSC precu...

    Authors: Chantal Lechanteur, Alexandra Briquet, Olivier Giet, Olivier Delloye, Etienne Baudoux and Yves Beguin
    Citation: Journal of Translational Medicine 2016 14:145
  5. The trophic, anti-apoptotic and regenerative effects of bone marrow mesenchymal stromal cells (MSC) may reduce neuronal cell loss in neurodegenerative disorders.

    Authors: Margherita Canesi, Rosaria Giordano, Lorenza Lazzari, Maurizio Isalberti, Ioannis Ugo Isaias, Riccardo Benti, Paolo Rampini, Giorgio Marotta, Aurora Colombo, Emanuele Cereda, Mariangela Dipaola, Tiziana Montemurro, Mariele Viganò, Silvia Budelli, Elisa Montelatici, Cristiana Lavazza…
    Citation: Journal of Translational Medicine 2016 14:127
  6. Adipose-derived stem cells (ASCs) have been identified as a population of multipotent cells with promising applications in tissue engineering and regenerative medicine. ASCs are abundant in fat tissue, which c...

    Authors: Dominik Duscher, Anna Luan, Robert C. Rennert, David Atashroo, Zeshaan N. Maan, Elizabeth A. Brett, Alexander J. Whittam, Natalie Ho, Michelle Lin, Michael S. Hu, Graham G. Walmsley, Raphael Wenny, Manfred Schmidt, Arndt F. Schilling, Hans-Günther Machens, Georg M. Huemer…
    Citation: Journal of Translational Medicine 2016 14:126
  7. The mechanisms underlying discrimination between “self” and “non-self”, a central immunological principle, require careful consideration in immune oncology therapeutics where eliciting anti-cancer immunity mus...

    Authors: Samuel C. Wagner, Neil H. Riordan, Thomas E. Ichim, Julia Szymanski, Hong Ma, Jesus A. Perez, Javier Lopez, Juan J. Plata-Munoz, Francisco Silva, Amit N. Patel and Santosh Kesari
    Citation: Journal of Translational Medicine 2016 14:90
  8. Automation of cell therapy manufacturing promises higher productivity of cell factories, more economical use of highly-trained (and costly) manufacturing staff, facilitation of processes requiring manufacturin...

    Authors: Christiane Hümmer, Carolin Poppe, Milica Bunos, Belinda Stock, Eva Wingenfeld, Volker Huppert, Juliane Stuth, Kristina Reck, Mike Essl, Erhard Seifried and Halvard Bonig
    Citation: Journal of Translational Medicine 2016 14:76
  9. High levels of pro-inflammatory cytokines in leukocyte- and platelet-rich plasma (L-PRP) may activate the nuclear factor κB (NF-κB) pathway to counter the beneficial effect of the growth factors on bone regene...

    Authors: Wenjing Yin, Xin Qi, Yuelei Zhang, Jiagen Sheng, Zhengliang Xu, Shicong Tao, Xuetao Xie, Xiaolin Li and Changqing Zhang
    Citation: Journal of Translational Medicine 2016 14:73
  10. Adjuvant imatinib is useful in patients with gastrointestinal stromal tumors (GIST) at high risk of recurrence. At present, the risk of recurrence is determined based on tumor size, mitotic rate, tumor site, a...

    Authors: Keith M. Skubitz, Kate Geschwind, Wayne W. Xu, Joseph S. Koopmeiners and Amy P. N. Skubitz
    Citation: Journal of Translational Medicine 2016 14:51
  11. Orthotopic liver transplantation is the only effective treatment for liver failure but limited with shortage of available donor organs. Recent studies show promising results of mesenchymal stem cells (MSCs)-ba...

    Authors: Biao Huang, Xixi Cheng, Huafeng Wang, Wenjing Huang, Zha la Ga hu, Dan Wang, Kai Zhang, Huan Zhang, Zhenyi Xue, Yurong Da, Ning Zhang, Yongcheng Hu, Zhi Yao, Liang Qiao, Fei Gao and Rongxin Zhang
    Citation: Journal of Translational Medicine 2016 14:45
  12. Mesenchymal stromal cells (MSCs) are adult stem cells able to give rise to bone, cartilage and fat cells. In addition, they possess immunomodulatory and immunosuppressive properties that are mainly mediated th...

    Authors: Marie-Theres Zeuner, Ketan Patel, Bernd Denecke, Bernd Giebel and Darius Widera
    Citation: Journal of Translational Medicine 2016 14:34
  13. Endometrial regenerative cells (ERCs) is an attractive novel type of adult mesenchymal stem cells that can be non-invasively obtained from menstrual blood and are easily replicated at a large scale without tum...

    Authors: Peng Sun, Jian Liu, Wenwen Li, Xiaoxi Xu, Xiangying Gu, HongYue Li, Hongqiu Han, Caigan Du and Hao Wang
    Citation: Journal of Translational Medicine 2016 14:28
  14. Craniomaxillofacial defects secondary to trauma, tumor resection, or congenital malformations are frequent unmet challenges, due to suboptimal alloplastic options and limited autologous tissues such as bone. S...

    Authors: Amy L. Strong, Ryan S. Hunter, Robert B. Jones, Annie C. Bowles, Maria F. Dutreil, Dina Gaupp, Daniel J. Hayes, Jeffrey M. Gimble, Benjamin Levi, Margaret A. McNulty and Bruce A. Bunnell
    Citation: Journal of Translational Medicine 2016 14:27
  15. Umbilical cord blood (UCB) is becoming an alternative cell source for hematopoietic stem cell transplantation (HSCT). However, umbilical cord blood transplantation (UCBT) has been severely limited by low and f...

    Authors: Deng-Mei Tian, Ying-Min Liang and Yong-Qing Zhang
    Citation: Journal of Translational Medicine 2016 14:5
  16. Glioblastoma (GBM) is the deadliest of brain tumors. Standard treatment for GBM is surgery, followed by combined radiation therapy and chemotherapy. Current therapy prolongs survival but does not offer a cure....

    Authors: Mark S. Kindy, Jin Yu, Hong Zhu, Michael T. Smith and Sebastiano Gattoni-Celli
    Citation: Journal of Translational Medicine 2016 14:1
  17. Bacterial and fungal infections continue to pose a major clinical challenge in patients with prolonged severe neutropenia after chemotherapy or hematopoietic stem cell transplantation (HSCT). With the advent o...

    Authors: Chiara Cugno, Sara Deola, Perla Filippini, David F. Stroncek and Sergio Rutella
    Citation: Journal of Translational Medicine 2015 13:362
  18. Pooled human platelet lysate (pHPL) is an efficient alternative to xenogenic supplements for ex vivo expansion of mesenchymal stem cells (MSCs) in clinical studies. Currently, porcine heparin is used in pHPL-s...

    Authors: Sandra Laner-Plamberger, Thomas Lener, Doris Schmid, Doris A. Streif, Tina Salzer, Michaela Öller, Cornelia Hauser-Kronberger, Thorsten Fischer, Volker R. Jacobs, Katharina Schallmoser, Mario Gimona and Eva Rohde
    Citation: Journal of Translational Medicine 2015 13:354
  19. Mesenchymal stromal cells (MSC) may serve as an attractive therapy in renal transplantation due to their immunosuppressive and reparative properties. While most studies have used autologous MSCs, allogeneic MS...

    Authors: Marlies E. J. Reinders, Geertje J. Dreyer, Jonna R. Bank, Helene Roelofs, Sebastiaan Heidt, Dave L. Roelen, Maarten L. Zandvliet, Volkert A. L. Huurman, Wim E. Fibbe, Cees van Kooten, Frans H. J. Claas, Ton J. Rabelink and Johan W. de Fijter
    Citation: Journal of Translational Medicine 2015 13:344
  20. Demonstrate the safety and effectiveness of highly purified CD133+ autologous stem cells in critical limb ischemia (CLI).

    Authors: Vittorio Arici, Cesare Perotti, Calliada Fabrizio, Claudia Del Fante, Franco Ragni, Francesco Alessandrino, Gianluca Viarengo, Michele Pagani, Alessia Moia, Carmine Tinelli and Antonio Bozzani
    Citation: Journal of Translational Medicine 2015 13:342
  21. Angiogenesis is essential for the growth and metastasis of solid tumors. The tumor endothelium exists in a state of chronic activation and proliferation, fueled by the tumor milieu where angiogenic mediators a...

    Authors: Samuel C. Wagner, Thomas E. Ichim, Hong Ma, Julia Szymanski, Jesus A. Perez, Javier Lopez, Vladimir Bogin, Amit N. Patel, Francisco M. Marincola and Santosh Kesari
    Citation: Journal of Translational Medicine 2015 13:340
  22. Emerging evidence indicates that mesenchymal stromal cells (MSCs) isolated from different tissue sources may be used in vivo as tissue restorative agents. To date, there is no evidence, however, on migration a...

    Authors: Hatixhe Latifi-Pupovci, Zyrafete Kuçi, Sibylle Wehner, Halvard Bönig, Ralf Lieberz, Thomas Klingebiel, Peter Bader and Selim Kuçi
    Citation: Journal of Translational Medicine 2015 13:315
  23. Within the last few years, it has become evident that LPS-preconditioned mesenchymal stromal cells (LPS pre-MSCs) show enhanced paracrine effects, including increased trophic support and improved regenerative ...

    Authors: Dongdong Ti, Haojie Hao, Chuan Tong, Jiejie Liu, Liang Dong, Jingxi Zheng, Yali Zhao, Huiling Liu, Xiaobing Fu and Weidong Han
    Citation: Journal of Translational Medicine 2015 13:308
  24. Skeletal myoblasts (SkMs) has provided a promising treatment for myocardial infarction (MI). Functioning as posttranscriptional regulators, microRNAs (miRNAs) play important roles in cardiac repairment and s...

    Authors: Qi Liu, Guo Qing Du, Zhi Tao Zhu, ChunYang Zhang, Xiao Wei Sun, Jing Jin Liu, Xia Li, Yong Shun Wang and Wen Juan Du
    Citation: Journal of Translational Medicine 2015 13:270
  25. Placental like alkaline phosphate (PLAP), an oncofetal antigen, is highly expressed in germ cell, cervical, ovarian and several other tumour types but minimally in normal tissues. The expression of a PLAP prom...

    Authors: Imran Khan, Mohammad Khalid Zakaria, Mukesh Kumar, Prashant Mani, Parthaprasad Chattopadhyay, Debi P Sarkar and Subrata Sinha
    Citation: Journal of Translational Medicine 2015 13:254

    The Erratum to this article has been published in Journal of Translational Medicine 2015 13:300

  26. Non-healing wounds can pose a medical challenge as in the case of vasculopathic venostasis resulting in a surgical ulcer. When traditional approaches to wound care fail, an amniotic patch (a dehydrated tissue ...

    Authors: Neil H Riordan, Ben A George, Troy B Chandler and Randall W McKenna
    Citation: Journal of Translational Medicine 2015 13:242
  27. Reactivation of latent viruses such as human cytomegalovirus (HCMV) after allogeneic hematopoietic stem cell transplantation (HSCT) results in high morbidity and mortality. Effective immunization against HCMV ...

    Authors: Bala Sai Sundarasetty, Stephan Kloess, Olaf Oberschmidt, Sonja Naundorf, Klaus Kuehlcke, Anusara Daenthanasanmak, Laura Gerasch, Constanca Figueiredo, Rainer Blasczyk, Eliana Ruggiero, Raffaele Fronza, Manfred Schmidt, Christof von Kalle, Michael Rothe, Arnold Ganser, Ulrike Koehl…
    Citation: Journal of Translational Medicine 2015 13:240
  28. The rapid clinical translation of mesenchymal stem cells (MSC) has resulted in the development of cell-based strategies for multiple indications. Unfortunately one major barrier to widespread implementation of...

    Authors: Neil H Riordan, Marialaura Madrigal, Jason Reneau, Kathya de Cupeiro, Natalia Jiménez, Sergio Ruiz, Nelsy Sanchez, Thomas E Ichim, Francisco Silva and Amit N Patel
    Citation: Journal of Translational Medicine 2015 13:232
  29. The intestinal stem cells (ISC) modulation and the role of circulating hematopoietic stem cells (HSC) in coeliac disease (CD) are poorly understood. Our aim was to investigate the longitudinal modifications in...

    Authors: Anna Chiara Piscaglia, Sergio Rutella, Lucrezia Laterza, Valentina Cesario, Mariachiara Campanale, Immacolata Alessia Cazzato, Gianluca Ianiro, Federico Barbaro, Luca Di Maurizio, Giuseppina Bonanno, Tonia Cenci, Giovanni Cammarota, Luigi Maria Larocca and Antonio Gasbarrini
    Citation: Journal of Translational Medicine 2015 13:220
  30. Mesenchymal stromal cells (MSCs) expanded in vitro have been proposed as a potential therapy for congenital or acquired skin defects in pediatrics. The aim of this pre-clinical study was to investigate the ef...

    Authors: Gloria Pelizzo, Maria Antonietta Avanzini, Antonia Icaro Cornaglia, Monica Osti, Piero Romano, Luigi Avolio, Rita Maccario, Massimo Dominici, Annalisa De Silvestri, Erika Andreatta, Federico Costanzo, Melissa Mantelli, Daniela Ingo, Serena Piccinno and Valeria Calcaterra
    Citation: Journal of Translational Medicine 2015 13:219
  31. Limited adjuvant treatment options exist for patients with high-risk surgically resected melanoma. This first-in-human study investigated the safety, tolerability and immunologic correlates of Melanoma GVAX, a...

    Authors: Evan J Lipson, William H Sharfman, Shuming Chen, Tracee L McMiller, Theresa S Pritchard, January T Salas, Susan Sartorius-Mergenthaler, Irwin Freed, Sowmya Ravi, Hao Wang, Brandon Luber, Janice Davis Sproul, Janis M Taube, Drew M Pardoll and Suzanne L Topalian
    Citation: Journal of Translational Medicine 2015 13:214
  32. Mesenchymal stem cells (MSCs) are regarded as a promising cell-based therapeutic tool for tendon repair. This study aimed to compare the different tenogenic differentiation capacities of the three types of MSC...

    Authors: Linghui Dai, Xiaoqing Hu, Xin Zhang, Jingxian Zhu, Jiying Zhang, Xin Fu, Xiaoning Duan, Yingfang Ao and Chunyan Zhou
    Citation: Journal of Translational Medicine 2015 13:200
  33. Myocardial infarction (MI) results in damaged heart tissue which can progress to severely reduce cardiac function, leading to death. Recent studies have injected dissociated, suspended cardiac cells into coron...

    Authors: Dolly Holt-Casper, Jeff M Theisen, Alonso P Moreno, Mark Warren, Francisco Silva, David W Grainger, David A Bull and Amit N Patel
    Citation: Journal of Translational Medicine 2015 13:194
  34. The adoptive transfer of allogeneic antiviral T lymphocytes derived from seropositive donors can safely and effectively reduce or prevent the clinical manifestation of viral infections or reactivations in immu...

    Authors: Sabine Tischer, Christoph Priesner, Hans-Gert Heuft, Lilia Goudeva, Wolfgang Mende, Marc Barthold, Stephan Kloeß, Lubomir Arseniev, Krasimira Aleksandrova, Britta Maecker-Kolhoff, Rainer Blasczyk, Ulrike Koehl and Britta Eiz-Vesper
    Citation: Journal of Translational Medicine 2014 12:336
  35. The present study evaluated the efficacy and safety of human embryonic stem cell (hESC) therapy in patients with CP.

    Authors: Geeta Shroff, Anupama Gupta and Jitender Kumar Barthakur
    Citation: Journal of Translational Medicine 2014 12:318

    The Erratum to this article has been published in Journal of Translational Medicine 2017 15:193

  36. Multipotent mesenchymal stem/stromal cells (MSC) including adipose-derived stromal cells (ADSC) have been successfully applied for cardiovascular diseases treatment. Their regenerative potential is considered ...

    Authors: Nina A Dzhoyashvili, Anastasia Yu Efimenko, Tatiana N Kochegura, Natalia I Kalinina, Natalia V Koptelova, Olga Yu Sukhareva, Marina V Shestakova, Renat S Akchurin, Vsevolod A Tkachuk and Yelena V Parfyonova
    Citation: Journal of Translational Medicine 2014 12:337
  37. Kidney transplantation has improved survival and quality of life for patients with end-stage renal disease. Despite excellent short-term results due to better and more potent immunosuppressive drugs, long-term...

    Authors: Marlies EJ Reinders, Jonna R Bank, Geertje J Dreyer, Helene Roelofs, Sebastian Heidt, Dave L Roelen, Volkert AL Huurman, Jan Lindeman, Cees van Kooten, Frans HJ Claas, Wim E Fibbe, Ton J Rabelink and Johan W de Fijter
    Citation: Journal of Translational Medicine 2014 12:331
  38. Although a variety of drugs have been used to treat the symptoms of rheumatoid arthritis (RA), none of them are able to cure the disease. Interferon β (IFN-β) has pleiotropic effects on RA, but whether it can ...

    Authors: Rong Zhao, Ni-Nan Chen, Xiao-Wei Zhou, Ping Miao, Chao-Ying Hu, Liu Qian, Qi-Wen Yu, Ji-Ying Zhang, Hong Nie, Xue-hua Chen, Pu Li, Rong Xu, Lian-Bo Xiao, Xin Zhang, Jian-Ren Liu and Dong-Qing Zhang
    Citation: Journal of Translational Medicine 2014 12:330
  39. Dendritic cells are currently under investigation for their ability to generate anti-cancer immune responses. No consensus has been reached as to the optimal method of dendritic cell vaccine preparation and is...

    Authors: Mayu O Frank, Julia Kaufman, Salina Parveen, Nathalie E Blachère, Dana E Orange and Robert B Darnell
    Citation: Journal of Translational Medicine 2014 12:338
  40. Endometriosis is a benign chronic gynecological disease that affects women of reproductive age, characterized by the presence of functional endometrial tissues outside the uterine cavity. GnRH agonists exhibit...

    Authors: Huinan Weng, Fenghua Liu, Shuiwang Hu, Li Li and Yifeng Wang
    Citation: Journal of Translational Medicine 2014 12:306
  41. MicroRNA-200c (miR-200c) is one of the short noncoding RNAs that play crucial roles in tumorigenesis and tumor progression. It also acts as considerable modulator in the process of epithelial-to-mesenchymal tr...

    Authors: Lei Liu, Mingning Qiu, Guobin Tan, Ziji Liang, Yue Qin, Lieqian Chen, Hege Chen and Jianjun Liu
    Citation: Journal of Translational Medicine 2014 12:305

    The Correction to this article has been published in Journal of Translational Medicine 2022 20:153

  42. Graphene is a flat monolayer of carbon atoms, arranged in a two-dimensional hexagonal structure, with extraordinary electrical, thermal, and physical properties. Moreover, the molecular structure of graphene c...

    Authors: Eriberto Bressan, Letizia Ferroni, Chiara Gardin, Luca Sbricoli, Luca Gobbato, Francesco Saverio Ludovichetti, Ilaria Tocco, Amedeo Carraro, Adriano Piattelli and Barbara Zavan
    Citation: Journal of Translational Medicine 2014 12:296
  43. We have previously reported that dephnetin is therapeutically effective in the treatment of rheumatoid arthritis (RA) in collagen-induced arthritis (CIA) rat model. However, the molecular mechanism and the eff...

    Authors: Kuanyong Shu, Nanzhen Kuang, Zhiqin Zhang, Ziling Hu, Yujuan Zhang, Yingyuan Fu and Weiping Min
    Citation: Journal of Translational Medicine 2014 12:287
  44. The mesenchymal stem cell (MSC) is being broadly studied in clinical trials. Contrary to the early paradigm of cell replacement and differentiation as a therapeutic mechanism of action, evidence is mounting th...

    Authors: Marialaura Madrigal, Kosagisharaf S Rao and Neil H Riordan
    Citation: Journal of Translational Medicine 2014 12:260
  45. Despite advancements in wound healing techniques and devices, new treatments are needed to improve therapeutic outcomes. This study aimed to evaluate the potential use of a new biomaterial engineered from huma...

    Authors: Yinxin Fu, Junjie Guan, Shangchun Guo, Fei Guo, Xin Niu, Qiang Liu, Changqing Zhang, Huarong Nie and Yang Wang
    Citation: Journal of Translational Medicine 2014 12:274
  46. Cardiovascular cell therapy represents a promising field, with several approaches currently being tested. The advanced therapy medicinal product (ATMP) for the ongoing METHOD clinical study ("Bone marrow deriv...

    Authors: Marina Radrizzani, Viviana Lo Cicero, Sabrina Soncin, Sara Bolis, Daniel Sürder, Tiziano Torre, Francesco Siclari, Tiziano Moccetti, Giuseppe Vassalli and Lucia Turchetto
    Citation: Journal of Translational Medicine 2014 12:276
  47. The deregulation of microRNAs has been reported to play a pivotal role in hepatocellular carcinoma (HCC). MiR-126-3p has been reported to be associated with poor prognosis in HCC. However the underlying mechan...

    Authors: Chengli Du, Zhen Lv, Linping Cao, Chaofeng Ding, Owusu-ansah K Gyabaah, Haiyang Xie, Lin Zhou, Jian Wu and Shusen Zheng
    Citation: Journal of Translational Medicine 2014 12:259
  48. Peripheral blood mononuclear cells (PBMC) concentrates collected by apheresis are frequently used as starting material for cellular therapies, but the cell of interest must often be isolated prior to initiatin...

    Authors: David F Stroncek, Vicki Fellowes, Chauha Pham, Hanh Khuu, Daniel H Fowler, Lauren V Wood and Marianna Sabatino
    Citation: Journal of Translational Medicine 2014 12:241

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