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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: Methodology

    The quality and safety of advanced therapy products must be maintained throughout their production and quality control cycle to ensure their final use in patients. We validated the cell count method according ...

    Authors: Monica Gunetti, Sara Castiglia, Deborah Rustichelli, Katia Mareschi, Fiorella Sanavio, Michela Muraro, Elena Signorino, Laura Castello, Ivana Ferrero and Franca Fagioli

    Citation: Journal of Translational Medicine 2012 10:112

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  2. Content type: Research

    Cerebral palsy is currently one of the major diseases that cause severe paralysis of the nervous system in children; approximately 9–30% of cerebral palsy patients are also visually impaired, for which no effe...

    Authors: Min Li, Aixue Yu, Fangfang Zhang, GuangHui Dai, Hongbin Cheng, Xiaodong Wang and Yihua An

    Citation: Journal of Translational Medicine 2012 10:100

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  3. Content type: Research

    In vitro fabricated tissue engineered vascular constructs could provide an alternative to conventional substitutes. A crucial factor for tissue engineering of vascular constructs is an appropriate cell source. Va...

    Authors: Bianca Polchow, Kati Kebbel, Gerno Schmiedeknecht, Anne Reichardt, Wolfgang Henrich, Roland Hetzer and Cora Lueders

    Citation: Journal of Translational Medicine 2012 10:98

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  4. Content type: Research

    RhoA-Rho kinase complex contributes to keep the cavernosus smooth muscle contracted and its inhibition is considered a potential strategy for the therapy of erectile dysfunction (ED).

    Authors: Fabio Guagnini, Mara Ferazzini, Marco Grasso, Salvatore Blanco and Tiziano Croci

    Citation: Journal of Translational Medicine 2012 10:59

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  5. Content type: Research

    We conducted a pilot study of the infusion of intravenous autologous cord blood (CB) in children with cerebral palsy (CP) to assess the safety and feasibility of the procedure as well as its potential efficacy...

    Authors: Young-Ho Lee, Kyung Vin Choi, Jin Hwa Moon, Hyun-Joo Jun, Hye-Ryeong Kang, Se-In Oh, Hyung Sun Kim, Jang Soo Um, Mi Jung Kim, Yun Young Choi, Young-Jun Lee, Hee-Jin Kim, Jong-Hwa Lee, Su Min Son, Soo-Jin Choi, Wonil Oh…

    Citation: Journal of Translational Medicine 2012 10:58

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  6. Content type: Research

    This study evaluated the effectiveness of treating pseudarthrosis in rats by using bone marrow cell suspensions or cultures of bone marrow mesenchymal stromal cells

    Authors: Manoel Luiz Ferreira, Paulo Cesar Silva, Lucas Henrique Alvarez Silva, Danielle Cabral Bonfim, Lucas Cristo Conilho Macedo Müller, Christiano Costa Espósito and Alberto Schanaider

    Citation: Journal of Translational Medicine 2012 10:51

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  7. Content type: Research

    Human adipose-derived mesenchymal stem cells (ADMSCs) may be ideal source of cells for intervertebral disc (IVD) regeneration, but the harsh chemical microenvironment of IVD may significantly influence the bio...

    Authors: Chengzhen Liang, Hao Li, Yiqing Tao, Xiaopeng Zhou, Fangcai Li, Gang Chen and Qixin Chen

    Citation: Journal of Translational Medicine 2012 10:49

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  8. Content type: Research

    A summit on cellular therapy for cancer discussed and presented advances related to the use of adoptive cellular therapy for melanoma and other cancers. The summit revealed that this field is advancing rapidly...

    Authors: David F Stroncek, Carolina Berger, Martin A Cheever, Richard W Childs, Mark E Dudley, Peter Flynn, Luca Gattinoni, James R Heath, Michael Kalos, Francesco M Marincola, Jeffrey S Miller, Gustavo Mostoslavsky, Daniel J Powell Jr, Mahendra Rao, Nicholas P Restifo, Steven A Rosenberg…

    Citation: Journal of Translational Medicine 2012 10:48

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  9. Content type: Research

    Hepatitis C virus (HCV) causes liver fibrosis that may lead to liver cirrhosis or hepatocellular carcinoma (HCC), and may partially depend on infecting viral genotype. HCV genotype 3a is being more common in A...

    Authors: Waqar Ahmad, Bushra Ijaz and Sajida Hassan

    Citation: Journal of Translational Medicine 2012 10:41

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  10. Content type: Research

    Diabetic patients are at increased risk for bacterial infections; these studies provide new insight into the role of the host defense complement system in controlling bacterial pathogens in hyperglycemic envir...

    Authors: Pamela S Hair, Charlene G Echague, Reuben D Rohn, Neel K Krishna, Julius O Nyalwidhe and Kenji M Cunnion

    Citation: Journal of Translational Medicine 2012 10:35

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  11. Content type: Methodology

    There has been significant progress in the last two decades on the design of chimeric antigen receptors (CAR) for adoptive immunotherapy targeting tumor-associated antigens. Structurally CARs consist of a sing...

    Authors: Zhili Zheng, Nachimuthu Chinnasamy and Richard A Morgan

    Citation: Journal of Translational Medicine 2012 10:29

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  12. Content type: Methodology

    Bone marrow stromal cells (BMSCs) are being used to treat a variety of conditions. For many applications a supply of cryopreserved products that can be used for acute therapy is needed. The establishment of a ...

    Authors: Marianna Sabatino, Jiaqiang Ren, Virginia David-Ocampo, Lee England, Michael McGann, Minh Tran, Sergei A Kuznetsov, Hanh Khuu, Arun Balakumaran, Harvey G Klein, Pamela G Robey and David F Stroncek

    Citation: Journal of Translational Medicine 2012 10:23

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  13. Content type: Research

    Osteoblast and adipocyte are derived from common mesenchymal progenitor cells. The bone loss of osteoporosis is associated with altered progenitor differentiation from an osteoblastic to an adipocytic lineage....

    Authors: Li You, Ling Pan, Lin Chen, Jin-Yu Chen, Xiaoping Zhang, Zhongwei Lv and Da Fu

    Citation: Journal of Translational Medicine 2012 10:11

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  14. Content type: Research

    For clinical applications, dendritic cells (DCs) need to be generated using GMP-approved reagents. In this study, we tested the characteristics of DCs generated in two clinical grade culture media and activate...

    Authors: Jitka Fučíková, Daniela Rožková, Hana Ulčová, Vít Budinský, Klára Sochorová, Kateřina Pokorná, Jiřina Bartůňková and Radek Špíšek

    Citation: Journal of Translational Medicine 2011 9:223

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  15. Content type: Research

    Breast cancer stem cells (BCSCs) are the source of breast tumors. Compared with other cancer cells, cancer stem cells show high resistance to both chemotherapy and radiotherapy. Targeting of BCSCs is thus a po...

    Authors: Phuc V Pham, Nhan LC Phan, Nhung T Nguyen, Nhung H Truong, Thuy T Duong, Dong V Le, Kiet D Truong and Ngoc K Phan

    Citation: Journal of Translational Medicine 2011 9:209

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  16. Content type: Research

    Erythropoietin (EPO) is a glycoprotein hormone which is required to regulate the production of red blood cells. Deficiency of EPO is known to cause anemia in chronically infected renal patients and they requir...

    Authors: Humera Kausar, Sana Gull, Bushra Ijaz, Waqar Ahmad, Muhammad Tahir Sarwar, Zafar Iqbal, Zafar Nawaz, Sheikh Riazuddin and Sajida Hassan

    Citation: Journal of Translational Medicine 2011 9:186

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  17. Content type: Research

    Chronic refractory angina is a challenging clinical problem with limited treatment options. The results of early cardiovascular stem cell trials using ABMMC have been promising but have utilized intracoronary ...

    Authors: Jorge Tuma, Roberto Fernández-Viña, Antonio Carrasco, Jorge Castillo, Carlos Cruz, Alvaro Carrillo, Jose Ercilla, Carlos Yarleque, Jaime Cunza, Timothy D Henry and Amit N Patel

    Citation: Journal of Translational Medicine 2011 9:183

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  18. Content type: Protocol

    Idiopathic pulmonary fibrosis represents a lethal form of progressive fibrotic lung disorder with gradually increasing incidence worldwide. Despite intense research efforts its pathogenesis is still elusive an...

    Authors: Argyris Tzouvelekis, George Koliakos, Paschalis Ntolios, Irene Baira, Evangelos Bouros, Anastasia Oikonomou, Athanassios Zissimopoulos, George Kolios, Despoina Kakagia, Vassilis Paspaliaris, Ioannis Kotsianidis, Marios Froudarakis and Demosthenes Bouros

    Citation: Journal of Translational Medicine 2011 9:182

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  19. Content type: Review

    Prolonged life expectancy, life style and environmental changes have caused a changing disease pattern in developed countries towards an increase of degenerative and autoimmune diseases. Stem cells have become...

    Authors: Jeong Chan Ra, Sung Keun Kang, Il Seob Shin, Hyeong Geun Park, Sang Aun Joo, Jeong Geun Kim, Byeong-Cheol Kang, Yong Soon Lee, Ken Nakama, Min Piao, Bertram Sohl and Andras Kurtz

    Citation: Journal of Translational Medicine 2011 9:181

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  20. Content type: Research

    Ten cancer patients (Six renal cell carcinoma and four breast cancer patients) were treated in a phase I/II study with a vaccine composed of autologous dendritic cells (DCs) and IL-2 to evaluate the DC vaccine...

    Authors: Soyoung Baek, Choung-Soo Kim, Sung-Bae Kim, Yong-man Kim, Seog-Woon Kwon, YongMan Kim, HyunSoo Kim and Hyunah Lee

    Citation: Journal of Translational Medicine 2011 9:178

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  21. Content type: Research

    This study tested the superiority of combined cyclosporine A (CsA)-erythropoietin (EPO) therapy compared with either one in limiting brain infarction area (BIA) and preserving neurological function in rat afte...

    Authors: Chun-Man Yuen, Cheuk-Kwan Sun, Yu-Chun Lin, Li-Teh Chang, Ying-Hsien Kao, Chia-Hung Yen, Yung-Lung Chen, Tzu-Hsien Tsai, Sarah Chua, Pei-Lin Shao, Steve Leu and Hon-Kan Yip

    Citation: Journal of Translational Medicine 2011 9:141

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  22. Content type: Research

    Chronic inflammation plays an important role in head and neck squamous cell carcinomas (HNSCC). This study addresses the impact of two single nucleotide polymorphisms (SNP) Asp299Gly and Thr399Ile of the toll-...

    Authors: Christoph Bergmann, Hagen S Bachmann, Agnes Bankfalvi, Ramin Lotfi, Carolin Pütter, Clarissa A Wild, Patrick J Schuler, Jens Greve, Thomas K Hoffmann, Stephan Lang, André Scherag and Götz F Lehnerdt

    Citation: Journal of Translational Medicine 2011 9:139

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  23. Content type: Research

    Cardiomyocyte calcium overloading has been implicated in the pathogenesis of Duchenne muscular dystrophy (DMD) heart disease. The cardiac isoform of sarcoplasmic reticulum calcium ATPase (SERCA2a) plays a majo...

    Authors: Jin-Hong Shin, Brian Bostick, Yongping Yue, Roger Hajjar and Dongsheng Duan

    Citation: Journal of Translational Medicine 2011 9:132

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  24. Content type: Protocol

    Liver transplantation is the definitive treatment for many end-stage liver diseases. However, the life-long immunosuppression needed to prevent graft rejection causes clinically significant side effects. Cellu...

    Authors: Felix C Popp, Barbara Fillenberg, Elke Eggenhofer, Philipp Renner, Johannes Dillmann, Volker Benseler, Andreas A Schnitzbauer, James Hutchinson, Robert Deans, Deborah Ladenheim, Cheryl A Graveen, Florian Zeman, Michael Koller, Martin J Hoogduijn, Edward K Geissler, Hans J Schlitt…

    Citation: Journal of Translational Medicine 2011 9:124

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  25. Content type: Research

    This study tested the hypothesis that autologous transplantation of adipose-derived mesenchymal stem cells (ADMSCs) can effectively attenuate acute pulmonary ischemia-reperfusion (IR) injury.

    Authors: Cheuk-Kwan Sun, Chia-Hung Yen, Yu-Chun Lin, Tzu-Hsien Tsai, Li-Teh Chang, Ying-Hsien Kao, Sarah Chua, Morgan Fu, Sheung-Fat Ko, Steve Leu and Hon-Kan Yip

    Citation: Journal of Translational Medicine 2011 9:118

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  26. Content type: Research

    Lipid metabolites and cytokines, including chemokines and growth factors, are the key regulators of immune cell function and differentiation, and thus, dysregulation of these regulators is associated with vari...

    Authors: Hyun Ok Kim, Han-Soo Kim, Jong-Chan Youn, Eui-Cheol Shin and Sungha Park

    Citation: Journal of Translational Medicine 2011 9:113

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  27. Content type: Review

    The field of cellular therapy of cancer is moving quickly and the issues involved with its advancement are complex and wide ranging. The growing clinical applications and success of adoptive cellular therapy o...

    Authors: Cornelis JM Melief, John J O'Shea and David F Stroncek

    Citation: Journal of Translational Medicine 2011 9:107

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  28. Content type: Research

    In our recent study, Periostin was up-regulated in prostate cancer(PCa) compared with benign prostate hyperplasia (BPH) by proteomics analysis of prostate biopsies. We investigated the effect of sliencing Peri...

    Authors: Chuanyu Sun, Xiaojun Zhao, Ke Xu, Jian Gong, Weiwei Liu, Weihong Ding, Yuancheng Gou, Guowei Xia and Qiang Ding

    Citation: Journal of Translational Medicine 2011 9:99

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  29. Content type: Research

    The F11 Receptor (F11R; aka JAM-A, JAM-1) is a cell adhesion protein present constitutively on the membrane surface of circulating platelets and within tight junctions of endothelial cells (ECs). Previous repo...

    Authors: Bani M Azari, Jonathan D Marmur, Moro O Salifu, Yigal H Ehrlich, Elizabeth Kornecki and Anna Babinska

    Citation: Journal of Translational Medicine 2011 9:98

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  30. Content type: Research

    Hemoglobin E/β-thalassemia is particularly common in Southeast Asia and has variable symptoms ranging from mild to severe anemia. Previous investigations demonstrated the remarkable symptoms of β-thalassemia i...

    Authors: Saranyoo Ponnikorn, Tasanee Panichakul, Kitima Sresanga, Chokdee Wongborisuth, Sittiruk Roytrakul, Suradej Hongeng and Sumalee Tungpradabkul

    Citation: Journal of Translational Medicine 2011 9:96

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  31. Content type: Research

    Reactive oxygen species are important mediators exerting toxic effects on various organs during ischemia-reperfusion (IR) injury. We hypothesized that adipose-derived mesenchymal stem cells (ADMSCs) protect th...

    Authors: Yen-Ta Chen, Cheuk-Kwan Sun, Yu-Chun Lin, Li-Teh Chang, Yung-Lung Chen, Tzu-Hsien Tsai, Sheng-Ying Chung, Sarah Chua, Ying-Hsien Kao, Chia-Hung Yen, Pei-Lin Shao, Kuan-Cheng Chang, Steve Leu and Hon-Kan Yip

    Citation: Journal of Translational Medicine 2011 9:51

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  32. Content type: Review

    Emerging evidence suggests that cancer stem cells account for the initiation and progression of cancer. While many types of cancer stem cells with specific markers have been isolated and identified, a variety ...

    Authors: Hai-Guang Liu, Chong Chen, Han Yang, Yi-Fei Pan and Xiao-Hua Zhang

    Citation: Journal of Translational Medicine 2011 9:50

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  33. Content type: Methodology

    siRNA technology is a promising tool for gene therapy of vascular disease. Due to the multitude of reagents and cell types, RNAi experiment optimization can be time-consuming. In this study adherent cell cytom...

    Authors: Christoph S Nabzdyk, Maggie Chun, Leena Pradhan and Frank W LoGerfo

    Citation: Journal of Translational Medicine 2011 9:48

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  34. Content type: Research

    Exosomes or secreted bi-lipid vesicles from human ESC-derived mesenchymal stem cells (hESC-MSCs) have been shown to reduce myocardial ischemia/reperfusion injury in animal models. However, as hESC-MSCs are not...

    Authors: Tian Sheng Chen, Fatih Arslan, Yijun Yin, Soon Sim Tan, Ruenn Chai Lai, Andre Boon Hwa Choo, Jayanthi Padmanabhan, Chuen Neng Lee, Dominique PV de Kleijn and Sai Kiang Lim

    Citation: Journal of Translational Medicine 2011 9:47

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  35. Content type: Research

    Polyethyleneimine (PEI), which can interact with negatively charged DNA through electrostatic interaction to form nanocomplexes, has been widely attempted to use as a gene delivery system. However, PEI has som...

    Authors: Zhizhong Xu, Guobo Shen, Xiangying Xia, Xinyu Zhao, Peng Zhang, Huanhuan Wu, Qingfa Guo, Zhiyong Qian, Yuquan Wei and Shufang Liang

    Citation: Journal of Translational Medicine 2011 9:46

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  36. Content type: Research

    Gene therapy is a promising therapeutic approach for cancer. Targeted expression of desired therapeutic proteins within the tumor is the best approach to reduce toxicity and improve survival. This study is to ...

    Authors: Yu Xu, Jinxuan Hou, Zhengchun Liu, Haijun Yu, Wenjie Sun, Jie Xiong, Zhengkai Liao, Fuxiang Zhou, Conghua Xie and Yunfeng Zhou

    Citation: Journal of Translational Medicine 2011 9:39

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  37. Content type: Research

    The aim of the present study was to analyze the expression of Cyclin-dependent kinase 4 (CDK4) in lung cancer and its correlation with clinicopathologic features. Furthermore, the involvement of CDK4-mediated cel...

    Authors: Aibing Wu, Bin Wu, Jinsong Guo, Weiren Luo, Dong Wu, Huiling Yang, Yan Zhen, Xiaoli Yu, Hao Wang, Ying Zhou, Zhen Liu, Weiyi Fang and Zhixiong Yang

    Citation: Journal of Translational Medicine 2011 9:38

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  38. Content type: Review

    Stem cell therapy holds the promise to treat degenerative diseases, cancer and repair of damaged tissues for which there are currently no or limited therapeutic options. The potential of stem cell therapies ha...

    Authors: Carla A Herberts, Marcel SG Kwa and Harm PH Hermsen

    Citation: Journal of Translational Medicine 2011 9:29

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  39. Content type: Erratum

    After publication of our article [1], we realized the need for posting a correction note in order to prevent i) overinterpretation of some results by the readers and ii) concerns about potentially unintended misg...

    Authors: Michaela Muth, Kais Hussein, Christoph Jacobi, Hans Kreipe and Oliver Bock

    Citation: Journal of Translational Medicine 2011 9:24

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    The original article was published in Journal of Translational Medicine 2010 8:33

  40. Content type: Commentary

    We challenge the hypothesis of enhanced myocardial reperfusion after implanting a left ventricular assist device together with bone marrow mononuclear stem cells in patients with end-stage ischemic cardiomyopa...

    Authors: Kyriakos Anastasiadis, Polychronis Antonitsis, Helena Argiriadou, Georgios Koliakos, Argyrios Doumas, Andre Khayat, Christos Papakonstantinou and Stephen Westaby

    Citation: Journal of Translational Medicine 2011 9:12

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  41. Content type: Research

    We investigated whether myocardium-derived conditioned medium (MDCM) is effective in preserving left ventricular (LV) function in a rat acute myocardial infarction (AMI) model.

    Authors: Steve Leu, Ying-Hsien Kao, Cheuk-Kwan Sun, Yu-Chun Lin, Tzu-Hsien Tsai, Li-Teh Chang, Sarah Chua, Kuo-Ho Yeh, Chiung-Jen Wu, Morgan Fu and Hon-Kan Yip

    Citation: Journal of Translational Medicine 2011 9:11

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  42. Content type: Research

    Mesenchymal stem cells derived from adipose tissue (ADSC) are multipotent stem cells, originated from the vascular-stromal compartment of fat tissue. ADSC are used as an alternative cell source for many differ...

    Authors: Anastasia Efimenko, Ekaterina Starostina, Natalia Kalinina and Alexandra Stolzing

    Citation: Journal of Translational Medicine 2011 9:10

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  43. Content type: Research

    Adenoid cystic carcinoma is one of the most common types of salivary gland cancers. The poor long-term prognosis for patients with adenoid cystic carcinoma is mainly due to local recurrence and distant metasta...

    Authors: Qin Xu, Xiuming Liu, Wantao Chen and Zhiyuan Zhang

    Citation: Journal of Translational Medicine 2010 8:136

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  44. Content type: Research

    Apoptosis is an early event involved in cardiomyopathy associated with diabetes mellitus. Toll-like receptor (TLR) signaling triggers cell apoptosis through multiple mechanisms. Up-regulation of TLR4 expressio...

    Authors: Yuwei Zhang, Tianqing Peng, Huaqing Zhu, Xiufen Zheng, Xusheng Zhang, Nan Jiang, Xiaoshu Cheng, Xiaoyan Lai, Aminah Shunnar, Manpreet Singh, Neil Riordan, Vladimir Bogin, Nanwei Tong and Wei-Ping Min

    Citation: Journal of Translational Medicine 2010 8:133

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  45. Content type: Research

    Growth hormone (GH) is an essential regulator of intrahepatic lipid metabolism by activating multiple complex hepatic signaling cascades. Here, we examined whether chronic exogenous GH administration (via gene...

    Authors: Ying Qin and Ya-ping Tian

    Citation: Journal of Translational Medicine 2010 8:120

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  46. Content type: Research

    Culturing otospheres from dissociated organ of Corti is an appropriate starting point aiming at the development of cell therapy for hair cell loss. Although guinea pigs have been widely used as an excellent ex...

    Authors: Jeanne Oiticica, Luiz Carlos M Barboza-Junior, Ana Carla Batissoco, Karina Lezirovitz, Regina C Mingroni-Netto, Luciana A Haddad and Ricardo F Bento

    Citation: Journal of Translational Medicine 2010 8:119

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  47. Content type: Commentary

    Recent years have seen a growing interest in insulin-like growth factor 1 receptor (IGF1R) in medical oncology. Interesting data have been reported also on IGF1r in gastrointestinal stromal tumors (GISTs) espe...

    Authors: Maria A Pantaleo, Annalisa Astolfi, Margherita Nannini and Guido Biasco

    Citation: Journal of Translational Medicine 2010 8:117

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  48. Content type: Research

    The ability to expand virus- or tumor-specific T cells without damaging their functional capabilities is critical for success adoptive transfer immunotherapy of patients with opportunistic infection or tumor. ...

    Authors: Yixin Li and Roger J Kurlander

    Citation: Journal of Translational Medicine 2010 8:104

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