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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

A therapeutic cancer vaccine against GL261 murine glioma
Mark S. Kindy, et al



Endothelium-targeted human Delta-like 1 enhances the regeneration and homing of human cord blood stem and progenitor cells
Deng-Mei Tian, Ying-Min Liang and Yong-Qing Zhang


Obesity inhibits the osteogenic differentiation of human adipose-derived stem cells
Amy L. Strong, et al

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  1. Content type: Research

    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

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  2. Content type: Review

    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

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  3. Content type: Research

    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

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  4. Content type: Research

    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

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  5. Content type: Research

    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

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  6. Content type: Research

    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

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  7. Content type: Research

    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

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  8. Content type: Research

    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

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  9. Content type: Research

    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

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  10. Content type: Research

    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

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  11. Content type: Research

    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

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  12. Content type: Review

    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

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  13. Content type: Research

    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

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  14. Content type: Research

    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

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  15. Content type: Research

    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

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  16. Content type: Research

    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

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  17. Content type: Research

    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

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  18. Content type: Research

    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

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  19. Content type: Research

    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

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  20. Content type: Research

    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

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  21. Content type: Research

    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

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  22. Content type: Research

    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

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  23. Content type: Research

    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

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  24. Content type: Research

    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

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  25. Content type: Research

    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

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  26. Content type: Research

    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

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  27. Content type: Research

    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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  28. Content type: Research

    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

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  29. Content type: Review

    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

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  30. Content type: Research

    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

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  31. Content type: Research

    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

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  32. Content type: Research

    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

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  33. Content type: Research

    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

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  34. Content type: Research

    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

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  35. Content type: Research

    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

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  36. Content type: Research

    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

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  37. Content type: Research

    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

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  38. Content type: Research

    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

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  39. Content type: Research

    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

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  40. Content type: Research

    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

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  41. Content type: Research

    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

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  42. Content type: Methodology

    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

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  43. Content type: Research

    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

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  44. Content type: Research

    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

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  45. Content type: Research

    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

    Published on:

  46. Content type: Research

    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

    Published on:

  47. Content type: Research

    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

    Published on:

  48. Content type: Research

    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

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