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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. 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
  2. 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
  3. 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

  4. 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
  5. 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
  6. 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
  7. 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
  8. 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
  9. 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

  10. 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
  11. 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
  12. 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
  13. 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
  14. 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
  15. 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
  16. 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
  17. At present, no effective clinical treatment is available for the late effects of radiation myelopathy. The aim of the present study was to assess the therapeutic effects of human umbilical cord-derived mesench...

    Authors: Li Wei, Jing Zhang, Xiu-Bin Xiao, Hai-Xing Mai, Ke Zheng, Wan-Liang Sun, Lei Wang, Feng Liang, Zai-Liang Yang, Yuan Liu, Yan-Qing Wang, Zhi-Fang Li, Jia-Ning Wang, Wei-Jing Zhang and Hua You
    Citation: Journal of Translational Medicine 2014 12:246
  18. Aberrant expression of miR-1 has been implicated in various cancers. However, the mechanisms underlying the role of miR-1 in CRC progression still have not been clarified clearly. Here, we showed the decreased...

    Authors: Lijun Xu, Yue Zhang, Hui Wang, Guanhua Zhang, Yanqing Ding and Liang Zhao
    Citation: Journal of Translational Medicine 2014 12:244
  19. The inadequacy of existing therapeutic tools together with the paucity of organ donors have always led medical researchers to innovate the current treatment methods or to discover new ways to cure disease. Eme...

    Authors: Thekkeparambil Chandrabose Srijaya, Thamil Selvee Ramasamy and Noor Hayaty Abu Kasim
    Citation: Journal of Translational Medicine 2014 12:243
  20. HLA-haploidentical hematopoietic stem cell transplantation (HSCT) is suitable for patients lacking related or unrelated HLA-matched donors. Herein, we investigated whether plerixafor (MZ), as an adjunct to G-C...

    Authors: Sergio Rutella, Perla Filippini, Valentina Bertaina, Giuseppina Li Pira, Lidia Altomare, Stefano Ceccarelli, Letizia P Brescia, Barbarella Lucarelli, Elia Girolami, Gianpiero Conflitti, Maria Giuseppina Cefalo, Alice Bertaina, Tiziana Corsetti, Lorenzo Moretta and Franco Locatelli
    Citation: Journal of Translational Medicine 2014 12:240
  21. Activation of the PI3K/AKT pathway, a common mechanism in all subtypes of endometrial cancers (endometrioid and non-endometrioid tumors), has important roles in contributing to epithelial-mesenchymal transitio...

    Authors: Peixin Dong, Yosuke Konno, Hidemichi Watari, Masayoshi Hosaka, Masayuki Noguchi and Noriaki Sakuragi
    Citation: Journal of Translational Medicine 2014 12:231
  22. High mobility group box protein 1 (HMGB1) acts as an endogenous danger molecule that is released from necrotic cells and activated macrophages. We have previously shown that peptide Hp91, whose sequence corres...

    Authors: Rebecca Saenz, Diahnn Futalan, Lien Leutenez, Fien Eekhout, Jessie F Fecteau, Simeon Sundelius, Stig Sundqvist, Marie Larsson, Tomoko Hayashi, Boris Minev, Dennis Carson, Sadik Esener, Bradley Messmer and Davorka Messmer
    Citation: Journal of Translational Medicine 2014 12:211
  23. Transcription factor Sp1 is multifaceted, with the ability to function as an oncogene or a tumor suppressor, depending on the cellular context. We previously reported that Sp1 is required for the transcription...

    Authors: Jing-Ping Zhang, Hua Zhang, Hong-Bo Wang, Yan-Xian Li, Gui-Hong Liu, Shan Xing, Man-Zhi Li and Mu-Sheng Zeng
    Citation: Journal of Translational Medicine 2014 12:222
  24. The biologic heterogeneity of soft tissue sarcomas (STS), even within histological subtypes, complicates treatment. In earlier studies, gene expression patterns that distinguish two subsets of clear cell renal...

    Authors: Keith M Skubitz, Amy PN Skubitz, Wayne W Xu, Xianghua Luo, Pauline Lagarde, Jean-Michel Coindre and Frédéric Chibon
    Citation: Journal of Translational Medicine 2014 12:176
  25. Human bone marrow mesenchymal stem cells (hBMSCs) are multipotent cells that can differentiate into a variety of cell types. Elevated expression of peroxisome proliferator-activated receptor-γ (PPARγ) promotes...

    Authors: Junkui Sun, Yisheng Wang, Yuebai Li and Guoqiang Zhao
    Citation: Journal of Translational Medicine 2014 12:168
  26. Rheumatoid arthritis (RA) is a debilitating and painful disease leading to increased morbidity and mortality and novel therapeutic approaches are needed. The purpose of this study was to elucidate if mesenchym...

    Authors: Oksana Kehoe, Alison Cartwright, Ayman Askari, Alicia J El Haj and Jim Middleton
    Citation: Journal of Translational Medicine 2014 12:157
  27. Previous reports showed the presence of limited numbers of stem cells in neonatal murine cochlear sensory epithelia and these cells are progressively lost during the postnatal development. The goal of this stu...

    Authors: Xiangxin Lou, Youyi Dong, Jing Xie, Xianliu Wang, Liangliang Yang, Masaaki Tokuda and Yanzhong Zhang
    Citation: Journal of Translational Medicine 2014 12:150
  28. While substantial progress has been made in blocking acute transplant rejection with the advent of immune suppressive drugs, chronic rejection, mediated primarily by recipient antigen presentation, remains a f...

    Authors: Xusheng Zhang, Yanling Liu, Guangfeng Zhang, Jun Shi, Xiao Zhang, Xiufen Zheng, Alex T Jiang, Zhu-Xu Zhang, Nathan Johnston, King Sun Siu, Ruiqi Chen, Dameng Lian, David Koos, Douglas Quan and Wei-Ping Min
    Citation: Journal of Translational Medicine 2014 12:142
  29. Diabetes mellitus (DM) is an incurable metabolic disease constituting a major threat to human health. Insulin-producing cells (IPCs) differentiated from mesenchymal stem cells (MSCs) hold great promise in the ...

    Authors: Huiting Qu, Xiaoli Liu, Yihong Ni, Yang Jiang, Xiaoli Feng, Juan Xiao, Yanan Guo, Dexiao Kong, Ai Li, Xiaomei Li, Xianghua Zhuang, Zhilun Wang, Yongjing Wang, Yali Chang, Shihong Chen, Feng Kong…
    Citation: Journal of Translational Medicine 2014 12:135
  30. The Forkhead box M1 (FOXM1), an important regulator of cell differentiation and proliferation, is overexpressed in a number of aggressive human carcinomas. The purpose of this study was to examine the expressi...

    Authors: Ning Wen, Yu Wang, Li Wen, Shu-Hua Zhao, Zhen-Hua Ai, Yi Wang, Bo Wu, Huai-Xiu Lu, Hong Yang, Wen-Chao Liu and Yu Li
    Citation: Journal of Translational Medicine 2014 12:134
  31. Interleukin-2 (IL-2) therapy has been demonstrated to induce responses in 10-20% of advanced melanoma and renal cell carcinoma patients, which translates into durable remissions in up to half of the responsers...

    Authors: Samuel C Wagner, Boris Markosian, Naseem Ajili, Brandon R Dolan, Andy J Kim, Doru T Alexandrescu, Constantin A Dasanu, Boris Minev, James Koropatnick, Francesco M Marincola and Neil H Riordan
    Citation: Journal of Translational Medicine 2014 12:127
  32. Bone tissue engineering is a new approach for the repair of orbital defects. The aim of the present study was to explore the feasibility of tissue-engineered bone constructed using bone marrow stromal cells (B...

    Authors: Yefei Wang, Xiaoping Bi, Huifang Zhou, Yuan Deng, Jing Sun, Caiwen Xiao, Ping Gu and Xianqun Fan
    Citation: Journal of Translational Medicine 2014 12:123
  33. Multiple myeloma is characterized by clonal expansion of B cells producing monoclonal immunoglobulins or fragments thereof, which can be detected in the serum and/or urine and are ideal target antigens for pat...

    Authors: Tim Roehnisch, Cornelia Then, Wolfgang Nagel, Christina Blumenthal, Todd Braciak, Mariel Donzeau, Thomas Böhm, Michael Flaig, Carole Bourquin and Fuat S Oduncu
    Citation: Journal of Translational Medicine 2014 12:119
  34. Autologous transplantation of modified mesenchymal stem cells (MSCs) is a promising candidate for the treatment of the refractory clinical disease, avascular necrosis of the femoral head (ANFH). Our previous a...

    Authors: Qian Wen, Chaoying Zhou, Wei Luo, Mingqian Zhou and Li Ma
    Citation: Journal of Translational Medicine 2014 12:114
  35. In osteosarcoma (OS) and most Ewing sarcoma (EWS) patients, the primary tumor originates in the bone. Although tumor resection surgery is commonly used to treat these diseases, it frequently leaves massive bon...

    Authors: Enrico Lucarelli, Chiara Bellotti, Melissa Mantelli, Maria Antonietta Avanzini, Rita Maccario, Francesca Novara, Giulia Arrigo, Orsetta Zuffardi, Monia Zuntini, Martina Pandolfi, Luca Sangiorgi, Daniela Lisini, Davide Donati and Serena Duchi
    Citation: Journal of Translational Medicine 2014 12:95
  36. Alpha fetoprotein (AFP) is an oncofetal antigen over-expressed by many hepatocellular cancers (HCC). We previously demonstrated that HLA-A2-restricted epitopes derived from AFP are immunogenic in vitro and in viv...

    Authors: Lisa H Butterfield, James S Economou, T Clark Gamblin and David A Geller
    Citation: Journal of Translational Medicine 2014 12:86
  37. To determine if a standardized, non-xenogenic, reduced manipulation cultivation and surgical transplantation of limbal stem cell grafts is a safe and effective treatment option for patients with total and part...

    Authors: Nadia Zakaria, Tine Possemiers, Sorcha Ní Dhubhghaill, Inge Leysen, Jos Rozema, Carina Koppen, Jean-Pierre Timmermans, Zwi Berneman and Marie-Jose Tassignon
    Citation: Journal of Translational Medicine 2014 12:58
  38. Mesenchymal stem cells (MSCs) are an attractive source of stem cells for clinical applications. These cells exhibit a multilineage differentiation potential and strong capacity for immune modulation. Thus, MSC...

    Authors: Phuc Van Pham, Ngoc Bich Vu, Vuong Minh Pham, Nhung Hai Truong, Truc Le-Buu Pham, Loan Thi-Tung Dang, Tam Thanh Nguyen, Anh Nguyen-Tu Bui and Ngoc Kim Phan
    Citation: Journal of Translational Medicine 2014 12:56
  39. According to cancer-related microRNA (miRNA) expression microarray research available in public databases, miR-362 expression is elevated in gastric cancer. However, the expression and biological role of miR-3...

    Authors: Jin-tang Xia, Lian-zhou Chen, Wei-hua Jian, Ke-Bing Wang, Yong-zhen Yang, Wei-ling He, Yu-long He, De Chen and Wen Li
    Citation: Journal of Translational Medicine 2014 12:33
  40. Supplements to support clinical-grade cultures of mesenchymal stem cells (MSC) are required to promote growth and expansion of these cells. Platelet lysate (PL) is a human blood component which may replace ani...

    Authors: Paola Iudicone, Daniela Fioravanti, Giuseppina Bonanno, Michelina Miceli, Claudio Lavorino, Pierangela Totta, Luigi Frati, Marianna Nuti and Luca Pierelli
    Citation: Journal of Translational Medicine 2014 12:28
  41. Effective methods for eradicating cancer stem cells (CSCs), which are highly tumorigenic and resistant to conventional therapies, are urgently needed. Our previous studies demonstrated that survivin-responsive...

    Authors: Kiyonori Tanoue, Yuqing Wang, Minako Ikeda, Kaoru Mitsui, Rie Irie, Takao Setoguchi, Setsuro Komiya, Shoji Natsugoe and Ken-ichiro Kosai
    Citation: Journal of Translational Medicine 2014 12:27
  42. Self-complementary adeno-associated virus (scAAV) vectors have become a desirable vector for therapeutic gene transfer due to their ability to produce greater levels of transgene than single-stranded AAV (ssAA...

    Authors: Geoffrey L Rogers, Ashley T Martino, Irene Zolotukhin, Hildegund CJ Ertl and Roland W Herzog
    Citation: Journal of Translational Medicine 2014 12:25
  43. Magnetic Resonance-guided High Intensity Focused Ultrasound (MRgHIFU) is a hybrid technology that aims to offer non-invasive thermal ablation of targeted tumors or other pathological tissues. Acoustic aberrati...

    Authors: Lorena Petrusca, Magalie Viallon, Romain Breguet, Sylvain Terraz, Gibran Manasseh, Vincent Auboiroux, Thomas Goget, Loredana Baboi, Patrick Gross, K Michael Sekins, Christoph D Becker and Rares Salomir
    Citation: Journal of Translational Medicine 2014 12:12
  44. It has been reported that the PI3K/AKT signaling pathway is activated in diffuse large B-cell lymphoma (DLBCL), PI3K constitutive activation plays a crucial role in PI3K/AKT pathway. However, the copy number v...

    Authors: Wenli Cui, Ying Cai, Weige Wang, Zebing Liu, Ping Wei, Rui Bi, Weixiang Chen, Menghong Sun and Xiaoyan Zhou
    Citation: Journal of Translational Medicine 2014 12:10
  45. Human adipose tissue is an ideal autologous source of mesenchymal stem cells (MSCs) for various regenerative medicine and tissue engineering strategies. Aged patients are one of the primary target populations ...

    Authors: Mahmood S Choudhery, Michael Badowski, Angela Muise, John Pierce and David T Harris
    Citation: Journal of Translational Medicine 2014 12:8
  46. Most tympanic membrane (TM) perforations heal spontaneously, but approximately 10-20% remain open as chronic TM perforations. Chronic perforations can lead to an impaired hearing ability and recurrent middle e...

    Authors: Yue Shen, Yongzhi Guo, Malgorzata Wilczynska, Jinan Li, Sten Hellström and Tor Ny
    Citation: Journal of Translational Medicine 2014 12:5

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