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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. 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
  2. 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
  3. 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
  4. 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
  5. 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
  6. 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
  7. 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
  8. 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
  9. 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
  10. 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
  11. 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
  12. 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
  13. E2A gene, which encodes two basic helix–loop–helix (bHLH) transcription factors E12 and E47, has been identified as regulator of B lymphoid hematopoiesis and suppressor of lymphoma. E47 protein was found to de...

    Authors: Hongchao Zhao, Ao Huang, Pu Li, Yingjun Quan, Bo Feng, Xuehua Chen, Zhihai Mao, Zhenggang Zhu and Minhua Zheng
    Citation: Journal of Translational Medicine 2013 11:317
  14. It is now 40 years since bisphosphonates (BPs) were first used in the clinic. So, it is timely to provide a brief review of what we have learned about these agents in bone disease. BPs are bone-specific and ha...

    Authors: Xiao-Long Xu, Wen-Long Gou, Ai-Yuan Wang, Yu Wang, Quan-Yi Guo, Qiang Lu, Shi-Bi Lu and Jiang Peng
    Citation: Journal of Translational Medicine 2013 11:303
  15. MicroRNAs (miRNAs) can act as either oncogenes or tumor suppressor genes under different conditions and thus can play a significant role in cancer development. We investigated miR-655 expression in a cohort of...

    Authors: Yuanyuan Wang, Wenqiao Zang, Yuwen Du, Yunyun Ma, Min Li, Ping Li, Xudong Chen, Tao Wang, Ziming Dong and Guoqiang Zhao
    Citation: Journal of Translational Medicine 2013 11:301

    The Retraction Note to this article has been published in Journal of Translational Medicine 2020 18:89

  16. B cell malignancies are characterized by clonal expansion of B cells expressing tumor-specific idiotypes on their surface. These idiotypes are ideal target antigens for an individualized immunotherapy. However...

    Authors: Tim Roehnisch, Cornelia Then, Wolfgang Nagel, Christina Blumenthal, Todd Braciak, Mariel Donzeau, Thomas Böhm, Carole Bourquin and Fuat Oduncu
    Citation: Journal of Translational Medicine 2013 11:267
  17. Gene therapy using a recombinant adenovirus (Ad) encoding secretory human endostatin (Ad-Endo) has been demonstrated to be a promising antiangiogenesis and antitumor strategy of in animal models and clinical t...

    Authors: Li-xia Li, Yan-ling Zhang, Ling Zhou, Miao-la Ke, Jie-min Chen, Xiang Fu, Chun-ling Ye, Jiang-xue Wu, Ran-yi Liu and Wenlin Huang
    Citation: Journal of Translational Medicine 2013 11:257
  18. To overcome the absorption of traditional fat grafting, techniques for adipose-derived regenerative cell (ADRC)-enriched fat grafting are currently being adapted for practical application. The Celution®800/CRS...

    Authors: Natsuko Kakudo, Yoshihito Tanaka, Naoki Morimoto, Takeshi Ogawa, Satoshi Kushida, Tomoya Hara and Kenji Kusumoto
    Citation: Journal of Translational Medicine 2013 11:254
  19. Clinical cardiac cell therapy using autologous somatic stem cells is restricted by age and disease-associated impairment of stem cell function. Juvenile cells possibly represent a more potent alternative, but ...

    Authors: Kristin Klose, Rajika Roy, Andreja Brodarac, Andreas Kurtz, Andrea Ode, Kyung-Sun Kang, Karen Bieback, Yeong-Hoon Choi and Christof Stamm
    Citation: Journal of Translational Medicine 2013 11:236
  20. Adipose-derived stromal cells (ASCs) stimulated with vascular endothelial growth factor (VEGF) and serum-deprived, are applied in the first in-man double-blind placebo-controlled MyStromalCell Trial, as a nove...

    Authors: Bjarke Follin, Josefine Tratwal, Mandana Haack-Sørensen, Jens Jørgen Elberg, Jens Kastrup and Annette Ekblond
    Citation: Journal of Translational Medicine 2013 11:219
  21. The brain cancer stem cell (CSC) model describes a small subset of glioma cells as being responsible for tumor initiation, conferring therapy resistance and tumor recurrence. In brain CSC, the PI3-K/AKT and the R...

    Authors: Klaus Mantwill, Ulrike Naumann, Janina Seznec, Vroni Girbinger, Hermann Lage, Pawel Surowiak, Dagmar Beier, Michel Mittelbronn, Jürgen Schlegel and Per Sonne Holm
    Citation: Journal of Translational Medicine 2013 11:216
  22. Gastric cancer is highly aggressive disease. Despite advances in diagnosis and therapy, the prognosis is still poor. Various genetic and molecular alterations are found in gastric cancer that underlies the mal...

    Authors: Javed Akhtar, Zhou Wang, Zhi Ping Zhang and Ming Ming Bi
    Citation: Journal of Translational Medicine 2013 11:212
  23. Therapeutic gene transfer is of significant value to elaborate efficient, durable treatments against human osteoarthritis (OA), a slow, progressive, and irreversible disorder for which there is no cure to date.

    Authors: Jagadeesh K Venkatesan, Ana Rey-Rico, Gertrud Schmitt, Anna Wezel, Henning Madry and Magali Cucchiarini
    Citation: Journal of Translational Medicine 2013 11:211
  24. The naked caspase-3 small interfering RNA (siRNA) infused into the renal artery during cold preservation was effective, but did not protect auto-transplant porcine kidneys with increased inflammation and apopt...

    Authors: Cheng Yang, Long Li, Yinjia Xue, Zitong Zhao, Tian Zhao, Yichen Jia, Ruiming Rong, Ming Xu, Michael L Nicholson, Tongyu Zhu and Bin Yang
    Citation: Journal of Translational Medicine 2013 11:210
  25. Ulcerative colitis (UC) is the most common form of inflammatory bowel disease in the USA. A key component of UC is the increase in inflammatory angiogenesis of the colon during active disease. This increase is...

    Authors: Walter E Cromer, Chaitanya V Ganta, Mihir Patel, James Traylor, Christopher G Kevil, J Steven Alexander and J Michael Mathis
    Citation: Journal of Translational Medicine 2013 11:207
  26. Longikaurin A is a natural ent-kaurene diterpenoid isolated from Isodon genus. The ent-kaurene diterpenoids isolated from medicinal plants have been shown to have anti-disease effects. The present study was desig...

    Authors: Qing-Feng Zou, Ji-Ke Du, Hua Zhang, Hong-Bo Wang, Ze-Dong Hu, Shu-Peng Chen, Yong Du, Man-Zhi Li, Dan Xie, Juan Zou, Han-Dong Sun, Jian-Xin Pu and Mu-Sheng Zeng
    Citation: Journal of Translational Medicine 2013 11:200
  27. The quality and safety of cell therapy products must be maintained throughout their production and quality control cycle, ensuring their final use in the patient. We validated the Lymulus Amebocyte Lysate (LAL...

    Authors: Deborah Rustichelli, Sara Castiglia, Monica Gunetti, Katia Mareschi, Elena Signorino, Michela Muraro, Laura Castello, Fiorella Sanavio, Marco Leone, Ivana Ferrero and Franca Fagioli
    Citation: Journal of Translational Medicine 2013 11:197
  28. Autism is a pervasive neurodevelopmental disorder. At present there are no defined mechanisms of pathogenesis and therapy is mostly limited to behavioral interventions. Stem cell transplantation may offer a un...

    Authors: Yong-Tao Lv, Yun Zhang, Min Liu, Jia-na-ti Qiuwaxi, Paul Ashwood, Sungho Charles Cho, Ying Huan, Ru-Cun Ge, Xing-Wang Chen, Zhao-Jing Wang, Byung-Jo Kim and Xiang Hu
    Citation: Journal of Translational Medicine 2013 11:196
  29. Oncolytic viruses have shown potential as cancer therapeutics, but not all patients seem to benefit from therapy. Polymorphisms in Fc gamma receptors (FcgRs) lead to altered binding affinity of IgG between the...

    Authors: Mari Hirvinen, Raita Heiskanen, Minna Oksanen, Saila Pesonen, Ilkka Liikanen, Timo Joensuu, Anna Kanerva, Vincenzo Cerullo and Akseli Hemminki
    Citation: Journal of Translational Medicine 2013 11:193
  30. The current study sought to investigate the safety of intraoperative and early postoperative continuous hyperthermic intraperitoneal perfusion (IEPCHIP) at different temperatures in a swine model of experiment...

    Authors: Sheng Li, Yong-lei Zhang, Jia-yuan Sun, Ya-wei Hua and Pei-hong Wu
    Citation: Journal of Translational Medicine 2013 11:181
  31. Adipose-derived stromal cells (ADSCs) are a good alternative to multipotent stem cells for regenerative medicine. Low tidal volume (LVT) has proved to be an effective ventilation strategy. However, it is not k...

    Authors: Zuo Di Liang, Xiu Ru Yin, Da Sheng Cai, Heng Zhou and Ling Pei
    Citation: Journal of Translational Medicine 2013 11:179
  32. Mesenchymal stem cells (MSCs) are known to be capable of suppressing inflammatory responses. We previously reported that intra-abdominal implantation of bone marrow-derived MSCs (BM-MSCs) sheet by laparotomy a...

    Authors: Xian-ming Fu, Aika Yamawaki-Ogata, Hideki Oshima, Yuichi Ueda, Akihiko Usui and Yuji Narita
    Citation: Journal of Translational Medicine 2013 11:175
  33. Regenerative medicine and particular adult stem cells represent an alternative option with several fruitful therapeutic applications in patients suffering from chronic lung diseases including idiopathic pulmon...

    Authors: Argyris Tzouvelekis, Vassilis Paspaliaris, George Koliakos, Paschalis Ntolios, Evangelos Bouros, Anastasia Oikonomou, Athanassios Zissimopoulos, Nikolaos Boussios, Brian Dardzinski, Dimitrios Gritzalis, Antonis Antoniadis, Marios Froudarakis, George Kolios and Demosthenes Bouros
    Citation: Journal of Translational Medicine 2013 11:171
  34. Glioblastoma multiforme (GBM) is one of the most aggressive forms of cancer with a high rate of recurrence. We propose a novel oncolytic vaccinia virus (VACV)-based therapy using expression of the bone morphog...

    Authors: Rohit Duggal, Ulrike Geissinger, Qian Zhang, Jason Aguilar, Nanhai G Chen, Elena Binda, Angelo L Vescovi and Aladar A Szalay
    Citation: Journal of Translational Medicine 2013 11:155
  35. Colorectal cancer (CRC) remains one of the most common cancers worldwide. We observed that MUC20 was significantly up-regulated in CRC patients with poor prognosis based on the microarray analysis. However, li...

    Authors: Xiuying Xiao, Lisha Wang, Ping Wei, Yayun Chi, Dali Li, Qifeng Wang, Shujuan Ni, Cong Tan, Weiqi Sheng, Menghong Sun, Xiaoyan Zhou and Xiang Du
    Citation: Journal of Translational Medicine 2013 11:151
  36. Peripheral vascular disease of the lower extremities comprises a clinical spectrum that extends from no symptoms to presentation with critical limb ischemia (CLI). Bone marrow derived Mesenchymal Stem Cells (B...

    Authors: Pawan K Gupta, Anoop Chullikana, Rajiv Parakh, Sanjay Desai, Anjan Das, Sanjay Gottipamula, Sagar Krishnamurthy, Naveen Anthony, Arun Pherwani and Anish S Majumdar
    Citation: Journal of Translational Medicine 2013 11:143
  37. The α2-adrenoreceptor agonist dexmedetomidine is known to provide renoprotection against ischemia and reperfusion (I/R) injury. However the underlying molecular mechanisms remain unclear. The purpose of this stud...

    Authors: Yanna Si, Hongguang Bao, Liu Han, Hongwei Shi, Yuan Zhang, Li Xu, Chenhui Liu, Jinsong Wang, Xiaobing Yang, Akbar Vohra and Daqing Ma
    Citation: Journal of Translational Medicine 2013 11:141
  38. While PDE5 inhibitors have revolutionized treatment of ED, approximately 30% of patients are non-responsive. A significant cause of this is vascular and smooth muscle dysfunction, as well as nerve atrophy. Aut...

    Authors: Thomas E Ichim, Timothy Warbington, Octav Cristea, Joseph L Chin and Amit N Patel
    Citation: Journal of Translational Medicine 2013 11:139
  39. Modified cell-based angiogenic therapy has become a promising novel strategy for ischemic heart and limb diseases. Most studies focused on myoblast, endothelial cell progenitors or bone marrow mesenchymal stro...

    Authors: Evgeny K Shevchenko, Pavel I Makarevich, Zoya I Tsokolaeva, Maria A Boldyreva, Veronika Yu Sysoeva, Vsevolod A Tkachuk and Yelena V Parfyonova
    Citation: Journal of Translational Medicine 2013 11:138
  40. In an attempt to engineer a regulatory compliant form of cell assisted lipotransfer in the U.S., the authors developed Autologous Fat Transfer with In-situ Mediation (AIM) for reconstruction of a refractory surgi...

    Authors: Allan Y Wu and David M Morrow
    Citation: Journal of Translational Medicine 2013 11:136

    The Erratum to this article has been published in Journal of Translational Medicine 2014 12:269

  41. Endothelial progenitor cells (EPCs) contribute to tumor angiogenesis and growth. We previously reported that over-expression of an inhibitor of DNA binding/differentiation 1 (Id1) in EPCs can enhance EPC proli...

    Authors: Yajuan Su, Lingjuan Gao, Lichen Teng, Ying Wang, Jialin Cui, Shiyun Peng and Songbin Fu
    Citation: Journal of Translational Medicine 2013 11:132
  42. Interactions between dendritic cells (DCs) and T cells play a critical role in the development of glomerulonephritis, which is a common cause of chronic kidney disease. DC-specific intercellular adhesion molec...

    Authors: Minchao Cai, Jing Wu, Chaoming Mao, Jianmin Ren, Pu Li, Xiao Li, Jiuchang Zhong, Chundi Xu and Tong Zhou
    Citation: Journal of Translational Medicine 2013 11:103
  43. Hepatopulmonary syndrome (HPS) is characterized by advanced liver disease, hypoxemia and intrapulmonary vascular dilatation (IPVD). The pathogenesis of HPS is not completely understood. Recent findings have es...

    Authors: Bin Yi, Jing Zeng, Guansong Wang, Guisheng Qian and Kaizhi Lu
    Citation: Journal of Translational Medicine 2013 11:98
  44. Mesenchymal stem cells (MSCs) are multi-potent non-hematopoietic progenitor cells possessing an immune-regulatory function, with suppression of proliferation of activated lymphocytes. In this study, adult livi...

    Authors: Hyunah Lee, Jae Berm Park, Sanghoon Lee, Soyoung Baek, HyunSoo Kim and Sung Joo Kim
    Citation: Journal of Translational Medicine 2013 11:96
  45. Celiac disease (CD) is mainly characterised by villous atrophy and mucosal architectural rearrangement. The fibroblasts (FBs) are the most abundant mesenchymal cell type in the intestinal mucosa and are respon...

    Authors: Leda Roncoroni, Luca Elli, Maria Teresa Bardella, Gianluca Perrucci, Michele Ciulla, Vincenza Lombardo, Carolina Tomba, Dario Conte and Luisa Doneda
    Citation: Journal of Translational Medicine 2013 11:91
  46. The use of adenoviral vector for gene therapy is still an important strategy for advanced cancers, however, the lack of the requisite coxsackie-adenovirus receptor in cancer cells and host immune response to a...

    Authors: Hua-shan Shi, Li-ping Yang, Wei Wei, Xiao-qing Su, Xiao-peng Li, Meng Li, Shun-tao Luo, Hai-long Zhang, Lian Lu, Yong-qiu Mao, Bing Kan and Li Yang
    Citation: Journal of Translational Medicine 2013 11:86
  47. Angiogenesis is a critical part of the endogenous repair process in brain injury and disease, and requires at least two sequential steps. First, angiogenic sprouting of endothelial cells occurs, which entails ...

    Authors: Mo Dao, Ciara C Tate, Michael McGrogan and Casey C Case
    Citation: Journal of Translational Medicine 2013 11:81
  48. Mesenchymal stem cell (MSC) transplantation has emerged as a promising therapy for liver fibrosis. Issues concerning poor MSC survival and engraftment in the fibrotic liver still persist and warrant developmen...

    Authors: Ghazanfar Ali Nasir, Sadia Mohsin, Mohsin Khan, Sulaiman Shams, Gibran Ali, Shaheen N Khan and Sheikh Riazuddin
    Citation: Journal of Translational Medicine 2013 11:78
  49. Heart failure is one of the key causes of morbidity and mortality world-wide. The recent findings that regeneration is possible in the heart have made stem cell therapeutics the Holy Grail of modern cardiovasc...

    Authors: Leo Bockeria, Vladimir Bogin, Olga Bockeria, Tatyana Le, Bagrat Alekyan, Erik J Woods, Amalia A Brown, Thomas E Ichim and Amit N Patel
    Citation: Journal of Translational Medicine 2013 11:56

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