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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. Cell therapies are being investigated as potential disease modifying treatment options for osteoarthritis (OA). Progenza (PRG) comprises in vitro expanded mesenchymal stem cells derived from human donor adipos...

    Authors: D. Kuah, S. Sivell, T. Longworth, K. James, A. Guermazi, F. Cicuttini, Y. Wang, S. Craig, G. Comin, D. Robinson and J. Wilson
    Citation: Journal of Translational Medicine 2018 16:49
  2. The role of transforming growth factorβ (TGF-β)-induced tumor progression in advanced malignancy is well established, but the involvement of long non-coding RNAs (lncRNAs) in TGF-β signaling remains unclear. T...

    Authors: Teng Huang, Wei Huang, Hong Lu, Bi-yun Zhang, Jun Ma, Di Zhao, Yi-jun Wang, Da-hai Yu and Xia He
    Citation: Journal of Translational Medicine 2018 16:46
  3. Critical tissue defects frequently result from trauma, burns, chronic wounds and/or surgery. The ideal treatment for such tissue loss is autografting, but donor sites are often limited. Tissue engineering (TE)...

    Authors: Pan Wu, Haojiao Chen, Ronghua Jin, Tingting Weng, Jon Kee Ho, Chuangang You, Liping Zhang, Xingang Wang and Chunmao Han
    Citation: Journal of Translational Medicine 2018 16:29
  4. We have recently shown that memory B cells from murine CMV immune donor animals adoptively transferred into immunodeficient mice were highly effective in protecting from a viral infection indicating a therapeu...

    Authors: Hannes Tittlbach, Andrea Schneider, Julian Strobel, Robert Zimmermann, Stefanie Maas, Bernd Gebhardt, Georg Rauser, Michael Mach, Andreas Mackensen, Thomas H. Winkler and Julia Winkler
    Citation: Journal of Translational Medicine 2017 15:228
  5. Degenerative disc disease (DDD) is a common cause of lower back pain with radicular symptoms and has a significant socioeconomic impact given the associated disability. Limited effective conservative therapeut...

    Authors: Christopher Centeno, Jason Markle, Ehren Dodson, Ian Stemper, Christopher J. Williams, Matthew Hyzy, Thomas Ichim and Michael Freeman
    Citation: Journal of Translational Medicine 2017 15:197
  6. Neurotrophins (NT) belongs to a family of growth factors which promotes neurons survival and differentiation. Increasing evidence show that NT and their receptor are expressed in lung tissues suggesting a poss...

    Authors: Emanuela Cherubini, Salvatore Mariotta, Davide Scozzi, Rita Mancini, Giorgia Osman, Michela D’Ascanio, Pierdonato Bruno, Giuseppe Cardillo and Alberto Ricci
    Citation: Journal of Translational Medicine 2017 15:196
  7. This review based on translational research predicts that the transcription factor p53 is the key effector of all anti-acne therapies. All-trans retinoic acid (ATRA) and isotretinoin (13-cis retinoic acid) enhanc...

    Authors: Bodo C. Melnik
    Citation: Journal of Translational Medicine 2017 15:195
  8. Reduced levels of endothelial progenitor cells (EPCs) counts have been reported in diabetic mellitus (DM) patients and other diabetes-related disorder. EPCs are a circulating, bone marrow-derived cell populati...

    Authors: Rashmi K. Ambasta, Harleen Kohli and Pravir Kumar
    Citation: Journal of Translational Medicine 2017 15:185
  9. In humans, the heterochronic cascade composed of the RNA-binding protein LIN28 and its major target, the let-7 family of microRNAs (miRNAs), is highly regulated during human erythroid ontogeny. Additionally, down...

    Authors: Jaira F. de Vasconcellos, Colleen Byrnes, Y. Terry Lee, Joshua M. Allwardt, Megha Kaushal, Antoinette Rabel and Jeffery L. Miller
    Citation: Journal of Translational Medicine 2017 15:169
  10. Cardiovascular disease remains a major health care challenge. The knowledge about the underlying mechanisms of the respective vascular disease etiologies has greatly expanded over the last decades. This includ...

    Authors: Christoph S. Nabzdyk, Leena Pradhan-Nabzdyk and Frank W. LoGerfo
    Citation: Journal of Translational Medicine 2017 15:164
  11. In an attempt to increase the therapeutic potential for myocardial regeneration, there is a quest for new cell sources and types for cell therapy protocols. The pathophysiology of heart diseases may affect cel...

    Authors: Lucinara Dadda Dias, Karina Rabello Casali, Carine Ghem, Melissa Kristocheck da Silva, Grasiele Sausen, Patrícia Bonini Palma, Dimas Tadeu Covas, Renato A. K. Kalil, Beatriz D. Schaan, Nance Beyer Nardi and Melissa Medeiros Markoski
    Citation: Journal of Translational Medicine 2017 15:161
  12. Cell-based therapy is being explored as an alternative treatment option for critical limb ischemia (CLI), a disease associated with high amputation and mortality rates and poor quality of life. However, therap...

    Authors: Adas Darinskas, Mindaugas Paskevicius, Gintaras Apanavicius, Gintaris Vilkevicius, Liutauras Labanauskas, Thomas E. Ichim and Rytis Rimdeika
    Citation: Journal of Translational Medicine 2017 15:143

    The Retraction Note to this article has been published in Journal of Translational Medicine 2020 18:90

  13. Stromal vascular fraction (SVF) can easily be obtained from a mini-lipoaspirate procedure of fat tissue and platelet rich plasma (PRP) can be obtained from peripheral blood. We evaluated the safety and prelimi...

    Authors: Himanshu Bansal, Kristin Comella, Jerry Leon, Poonam Verma, Diwaker Agrawal, Prasad Koka and Thomas Ichim
    Citation: Journal of Translational Medicine 2017 15:141

    The Retraction Note to this article has been published in Journal of Translational Medicine 2021 19:168

  14. Children with congenital heart defects (CHDs) are at high risk for myocardial failure after operative procedures with cardiopulmonary bypass (CPB). Recent studies suggest that microRNAs (miRNA) are involved in...

    Authors: Masood Abu-Halima, Martin Poryo, Nicole Ludwig, Janine Mark, Ina Marsollek, Christian Giebels, Johannes Petersen, Hans-Joachim Schäfers, Ulrich Grundmann, Thomas Pickardt, Andreas Keller, Eckart Meese and Hashim Abdul-Khaliq
    Citation: Journal of Translational Medicine 2017 15:117
  15. Numerous studies have investigated the role of the dietary factors in the prevention of cognitive decline but the short-term effects of foods choice on cognitive performances in the elderly are poorly explored...

    Authors: Elisa Mazza, Antonietta Fava, Yvelise Ferro, Marta Moraca, Stefania Rotundo, Carmela Colica, Francesco Provenzano, Rosa Terracciano, Marta Greco, Daniela Foti, Elio Gulletta, Diego Russo, Domenico Bosco, Arturo Pujia and Tiziana Montalcini
    Citation: Journal of Translational Medicine 2017 15:109
  16. Retinal degeneration (RD), such as age-related macular degeneration (AMD) and retinitis pigmentosa, is one of the leading causes of blindness. Presently, no satisfactory therapeutic options are available for t...

    Authors: Zhimin Tang, Yi Zhang, Yuyao Wang, Dandan Zhang, Bingqiao Shen, Min Luo and Ping Gu
    Citation: Journal of Translational Medicine 2017 15:99
  17. Adeno-associated virus (AAV) gene therapy vectors have shown the best outcomes in human clinical studies for the treatment of genetic diseases such as hemophilia. However, these pivotal investigations have als...

    Authors: David M. Markusic, Timothy C. Nichols, Elizabeth P. Merricks, Brett Palaschak, Irene Zolotukhin, Damien Marsic, Sergei Zolotukhin, Arun Srivastava and Roland W. Herzog
    Citation: Journal of Translational Medicine 2017 15:94
  18. The use of fetal bovine serum (FBS) as a media supplement for the ex vivo expansion of bone-marrow derived mesenchymal stromal cells (BM-MSC) has been discouraged by regulatory agencies, due to the risk of tra...

    Authors: Martina Bernardi, Francesco Agostini, Katia Chieregato, Eliana Amati, Cristina Durante, Mario Rassu, Marco Ruggeri, Sabrina Sella, Elisabetta Lombardi, Mario Mazzucato and Giuseppe Astori
    Citation: Journal of Translational Medicine 2017 15:90
  19. Hepatocellular carcinoma (HCC) is one of the most common cancers in China and frequently occurs with chronic hepatitis B virus infection. To investigate whether cell-based cancer immunotherapy induces tumor sp...

    Authors: Yanyan Han, Yeting Wu, Chou Yang, Jing Huang, Yabing Guo, Li Liu, Ping Chen, Dongyun Wu, Junyun Liu, Jin Li, Xiangjun Zhou and Jinlin Hou
    Citation: Journal of Translational Medicine 2017 15:64
  20. Clinical trials of Chimeric Antigen Receptor (CAR) T cells manufactured from autologous peripheral blood mononuclear cell (PBMC) concentrates for the treatment of hematologic malignancies have been promising, ...

    Authors: David F. Stroncek, Daniel W. Lee, Jiaqiang Ren, Marianna Sabatino, Steven Highfill, Hanh Khuu, Nirali N. Shah, Rosandra N. Kaplan, Terry J. Fry and Crystal L. Mackall
    Citation: Journal of Translational Medicine 2017 15:59
  21. Critical limb ischemia (CLI) causes severe ischemic rest pain, ulcer, and gangrene in the lower limbs. In spite of angioplasty and surgery, CLI patients without suitable artery inflow or enough vascular bed in...

    Authors: Makoto Samura, Tohru Hosoyama, Yuriko Takeuchi, Koji Ueno, Noriyasu Morikage and Kimikazu Hamano
    Citation: Journal of Translational Medicine 2017 15:49
  22. Bone marrow mesenchymal stem cells (BMMSCs) and bone marrow mononuclear cells (BMMNCs) are both used to treat spastic cerebral palsy. However, the differences in therapeutic effect remain unknown.

    Authors: Xuebin Liu, Xiaojun Fu, Guanghui Dai, Xiaodong Wang, Zan Zhang, Hongbin Cheng, Pei Zheng and Yihua An
    Citation: Journal of Translational Medicine 2017 15:48
  23. Sarcopenia, osteoporosis and osteoarthritis are the most frequent musculoskeletal disorders affecting older people. The main aim of this study was to test the hypothesis that the balance between BMPs and myost...

    Authors: Manuel Scimeca, Eleonora Piccirilli, Francesca Mastrangeli, Cecilia Rao, Maurizio Feola, Augusto Orlandi, Elena Gasbarra, Elena Bonanno and Umberto Tarantino
    Citation: Journal of Translational Medicine 2017 15:34
  24. Mesenchymal stem cell (MSC) has been widely explored in the past decade as a cell-based treatment for various diseases. However, poor survival of adaptively transferred MSCs limits their clinical therapeutic p...

    Authors: Shuo Wang, Chao Zhang, Sidikejiang Niyazi, Long Zheng, Jiawei Li, Weitao Zhang, Ming Xu, Ruiming Rong, Cheng Yang and Tongyu Zhu
    Citation: Journal of Translational Medicine 2017 15:33
  25. We performed a randomized, double-blind, cross-over study to assess the neuroregenerative potential of intravenous granulocyte colony-stimulating factor (G-CSF) followed by infusion of mobilized peripheral blo...

    Authors: Wee-Jin Rah, Young-Ho Lee, Jin-Hwa Moon, Hyun-Ju Jun, Hye-Ryeong Kang, Hani Koh, Hye Jung Eom, Ji Young Lee, Young Jun Lee, Ji Young Kim, Yun-Young Choi, Kyeongil Park, Mi Jung Kim and Seung-Hyun Kim
    Citation: Journal of Translational Medicine 2017 15:16
  26. Stromal vascular fraction (SVF) can easily be obtained from a mini-lipoaspirate procedure of fat tissue and platelet rich plasma (PRP) can be obtained from peripheral blood. The SVF contains a mixture of cells...

    Authors: Kristin Comella, Robert Silbert and Michelle Parlo
    Citation: Journal of Translational Medicine 2017 15:12

    The Erratum to this article has been published in Journal of Translational Medicine 2017 15:108

  27. The repair of urinary bladder tissue is a necessity for tissue loss due to cancer, trauma, or congenital abnormalities. Use of intestinal tissue is still the gold standard in the urological clinic, which leads...

    Authors: Dorothea Leonhäuser, Katja Stollenwerk, Volker Seifarth, Isabella M. Zraik, Michael Vogt, Pramod K. Srinivasan, Rene H. Tolba and Joachim O. Grosse
    Citation: Journal of Translational Medicine 2017 15:3
  28. Induced pluripotent stem cells (iPSC) possess an enormous potential as both, scientific and therapeutic tools. Their application in the regenerative medicine provides new treatment opportunities for numerous d...

    Authors: Maciej P. Walczak, Anna M. Drozd, Ewelina Stoczynska-Fidelus, Piotr Rieske and Dawid P. Grzela
    Citation: Journal of Translational Medicine 2016 14:341
  29. Recent studies suggest that immunotherapy using T regulatory cells (Tregs) prolongs remission in type 1 diabetes (T1DM). Here, we report factors that possibly affect the efficacy of this treatment.

    Authors: Natalia Marek-Trzonkowska, Małgorzata Myśliwiec, Dorota Iwaszkiewicz-Grześ, Mateusz Gliwiński, Ilona Derkowska, Magdalena Żalińska, Maciej Zieliński, Marcelina Grabowska, Hanna Zielińska, Karolina Piekarska, Anna Jaźwińska-Curyłło, Radosław Owczuk, Agnieszka Szadkowska, Krystyna Wyka, Piotr Witkowski, Wojciech Młynarski…
    Citation: Journal of Translational Medicine 2016 14:332
  30. Myocardial recovery with left ventricular assist device (LVAD) therapy is highly variable and difficult to predict. Next generation ribonucleic acid (RNA) sequencing is an innovative, rapid, and quantitative a...

    Authors: Kajari Dhar, Alexandra M. Moulton, Eric Rome, Fang Qiu, Jeff Kittrell, Eugenia Raichlin, Ronald Zolty, John Y. Um, Michael J. Moulton, Hesham Basma, Daniel R. Anderson, James D. Eudy and Brian D. Lowes
    Citation: Journal of Translational Medicine 2016 14:327
  31. Adipose derived stromal cells (ASCs) are a rich and convenient source of cells for clinical regenerative therapeutic approaches. However, applications of ASCs often require cell expansion to reach the needed d...

    Authors: Mandana Haack-Sørensen, Bjarke Follin, Morten Juhl, Sonja K. Brorsen, Rebekka H. Søndergaard, Jens Kastrup and Annette Ekblond
    Citation: Journal of Translational Medicine 2016 14:319
  32. Cell-based therapies such as autologous chondrocyte implantation are promising therapeutic approaches to treat cartilage defects to prevent further cartilage degeneration. To assure consistent quality of cell-...

    Authors: Christoph Bartz, Miriam Meixner, Petra Giesemann, Giulietta Roël, Grit-Carsta Bulwin and Jeske J. Smink
    Citation: Journal of Translational Medicine 2016 14:317
  33. The endometrial regenerative cell (ERC) is a novel type of adult mesenchymal stem cell isolated from menstrual blood. Previous studies demonstrated that ERCs possess unique immunoregulatory properties in vitro...

    Authors: Shanzheng Lu, Ganggang Shi, Xiaoxi Xu, Grace Wang, Xu Lan, Peng Sun, Xiang Li, Baoren Zhang, Xiangying Gu, Thomas E. Ichim and Hao Wang
    Citation: Journal of Translational Medicine 2016 14:300
  34. Gene therapy protocols require robust and long-term gene expression. For two decades, retrovirus family vectors have offered several attractive properties as stable gene-delivery vehicles. These vectors repres...

    Authors: José Eduardo Vargas, Leonardo Chicaybam, Renato Tetelbom Stein, Amilcar Tanuri, Andrés Delgado-Cañedo and Martin H. Bonamino
    Citation: Journal of Translational Medicine 2016 14:288
  35. 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
  36. 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
  37. 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
  38. 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
  39. 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
  40. 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
  41. 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
  42. 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
  43. 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
  44. 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
  45. 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
  46. 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
  47. 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
  48. 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

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