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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.

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  1. Content type: Review

    Reduced levels of endothelial progenitor cells (EPCs) counts have been reported in diabetic mellitus (DM) patients and other diabetes-related disorder. EPCs are a circulating, bone marrow-derived cell populati...

    Authors: Rashmi K. Ambasta, Harleen Kohli and Pravir Kumar

    Citation: Journal of Translational Medicine 2017 15:185

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  2. Content type: Research

    In humans, the heterochronic cascade composed of the RNA-binding protein LIN28 and its major target, the let-7 family of microRNAs (miRNAs), is highly regulated during human erythroid ontogeny. Additionally, down...

    Authors: Jaira F. de Vasconcellos, Colleen Byrnes, Y. Terry Lee, Joshua M. Allwardt, Megha Kaushal, Antoinette Rabel and Jeffery L. Miller

    Citation: Journal of Translational Medicine 2017 15:169

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  3. Content type: Review

    Cardiovascular disease remains a major health care challenge. The knowledge about the underlying mechanisms of the respective vascular disease etiologies has greatly expanded over the last decades. This includ...

    Authors: Christoph S. Nabzdyk, Leena Pradhan-Nabzdyk and Frank W. LoGerfo

    Citation: Journal of Translational Medicine 2017 15:164

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  4. Content type: Research

    In an attempt to increase the therapeutic potential for myocardial regeneration, there is a quest for new cell sources and types for cell therapy protocols. The pathophysiology of heart diseases may affect cel...

    Authors: Lucinara Dadda Dias, Karina Rabello Casali, Carine Ghem, Melissa Kristocheck da Silva, Grasiele Sausen, Patrícia Bonini Palma, Dimas Tadeu Covas, Renato A. K. Kalil, Beatriz D. Schaan, Nance Beyer Nardi and Melissa Medeiros Markoski

    Citation: Journal of Translational Medicine 2017 15:161

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  5. Content type: Research

    Cell-based therapy is being explored as an alternative treatment option for critical limb ischemia (CLI), a disease associated with high amputation and mortality rates and poor quality of life. However, therap...

    Authors: Adas Darinskas, Mindaugas Paskevicius, Gintaras Apanavicius, Gintaris Vilkevicius, Liutauras Labanauskas, Thomas E. Ichim and Rytis Rimdeika

    Citation: Journal of Translational Medicine 2017 15:143

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  6. Content type: Research

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

    Authors: Himanshu Bansal, Kristin Comella, Jerry Leon, Poonam Verma, Diwaker Agrawal, Prasad Koka and Thomas Ichim

    Citation: Journal of Translational Medicine 2017 15:141

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  7. Content type: Research

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

    Authors: Masood Abu-Halima, Martin Poryo, Nicole Ludwig, Janine Mark, Ina Marsollek, Christian Giebels, Johannes Petersen, Hans-Joachim Schäfers, Ulrich Grundmann, Thomas Pickardt, Andreas Keller, Eckart Meese and Hashim Abdul-Khaliq

    Citation: Journal of Translational Medicine 2017 15:117

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  8. Content type: Research

    Numerous studies have investigated the role of the dietary factors in the prevention of cognitive decline but the short-term effects of foods choice on cognitive performances in the elderly are poorly explored...

    Authors: Elisa Mazza, Antonietta Fava, Yvelise Ferro, Marta Moraca, Stefania Rotundo, Carmela Colica, Francesco Provenzano, Rosa Terracciano, Marta Greco, Daniela Foti, Elio Gulletta, Diego Russo, Domenico Bosco, Arturo Pujia and Tiziana Montalcini

    Citation: Journal of Translational Medicine 2017 15:109

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  9. Content type: Review

    Retinal degeneration (RD), such as age-related macular degeneration (AMD) and retinitis pigmentosa, is one of the leading causes of blindness. Presently, no satisfactory therapeutic options are available for t...

    Authors: Zhimin Tang, Yi Zhang, Yuyao Wang, Dandan Zhang, Bingqiao Shen, Min Luo and Ping Gu

    Citation: Journal of Translational Medicine 2017 15:99

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  10. Content type: Research

    Adeno-associated virus (AAV) gene therapy vectors have shown the best outcomes in human clinical studies for the treatment of genetic diseases such as hemophilia. However, these pivotal investigations have als...

    Authors: David M. Markusic, Timothy C. Nichols, Elizabeth P. Merricks, Brett Palaschak, Irene Zolotukhin, Damien Marsic, Sergei Zolotukhin, Arun Srivastava and Roland W. Herzog

    Citation: Journal of Translational Medicine 2017 15:94

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  11. Content type: Research

    The use of fetal bovine serum (FBS) as a media supplement for the ex vivo expansion of bone-marrow derived mesenchymal stromal cells (BM-MSC) has been discouraged by regulatory agencies, due to the risk of tra...

    Authors: Martina Bernardi, Francesco Agostini, Katia Chieregato, Eliana Amati, Cristina Durante, Mario Rassu, Marco Ruggeri, Sabrina Sella, Elisabetta Lombardi, Mario Mazzucato and Giuseppe Astori

    Citation: Journal of Translational Medicine 2017 15:90

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  12. Content type: Research

    Hepatocellular carcinoma (HCC) is one of the most common cancers in China and frequently occurs with chronic hepatitis B virus infection. To investigate whether cell-based cancer immunotherapy induces tumor sp...

    Authors: Yanyan Han, Yeting Wu, Chou Yang, Jing Huang, Yabing Guo, Li Liu, Ping Chen, Dongyun Wu, Junyun Liu, Jin Li, Xiangjun Zhou and Jinlin Hou

    Citation: Journal of Translational Medicine 2017 15:64

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  13. Content type: Research

    Clinical trials of Chimeric Antigen Receptor (CAR) T cells manufactured from autologous peripheral blood mononuclear cell (PBMC) concentrates for the treatment of hematologic malignancies have been promising, ...

    Authors: David F. Stroncek, Daniel W. Lee, Jiaqiang Ren, Marianna Sabatino, Steven Highfill, Hanh Khuu, Nirali N. Shah, Rosandra N. Kaplan, Terry J. Fry and Crystal L. Mackall

    Citation: Journal of Translational Medicine 2017 15:59

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  14. Content type: Review

    Critical limb ischemia (CLI) causes severe ischemic rest pain, ulcer, and gangrene in the lower limbs. In spite of angioplasty and surgery, CLI patients without suitable artery inflow or enough vascular bed in...

    Authors: Makoto Samura, Tohru Hosoyama, Yuriko Takeuchi, Koji Ueno, Noriyasu Morikage and Kimikazu Hamano

    Citation: Journal of Translational Medicine 2017 15:49

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  15. Content type: Research

    Bone marrow mesenchymal stem cells (BMMSCs) and bone marrow mononuclear cells (BMMNCs) are both used to treat spastic cerebral palsy. However, the differences in therapeutic effect remain unknown.

    Authors: Xuebin Liu, Xiaojun Fu, Guanghui Dai, Xiaodong Wang, Zan Zhang, Hongbin Cheng, Pei Zheng and Yihua An

    Citation: Journal of Translational Medicine 2017 15:48

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  16. Content type: Research

    Sarcopenia, osteoporosis and osteoarthritis are the most frequent musculoskeletal disorders affecting older people. The main aim of this study was to test the hypothesis that the balance between BMPs and myost...

    Authors: Manuel Scimeca, Eleonora Piccirilli, Francesca Mastrangeli, Cecilia Rao, Maurizio Feola, Augusto Orlandi, Elena Gasbarra, Elena Bonanno and Umberto Tarantino

    Citation: Journal of Translational Medicine 2017 15:34

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  17. Content type: Research

    Mesenchymal stem cell (MSC) has been widely explored in the past decade as a cell-based treatment for various diseases. However, poor survival of adaptively transferred MSCs limits their clinical therapeutic p...

    Authors: Shuo Wang, Chao Zhang, Sidikejiang Niyazi, Long Zheng, Jiawei Li, Weitao Zhang, Ming Xu, Ruiming Rong, Cheng Yang and Tongyu Zhu

    Citation: Journal of Translational Medicine 2017 15:33

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  18. Content type: Research

    We performed a randomized, double-blind, cross-over study to assess the neuroregenerative potential of intravenous granulocyte colony-stimulating factor (G-CSF) followed by infusion of mobilized peripheral blo...

    Authors: Wee-Jin Rah, Young-Ho Lee, Jin-Hwa Moon, Hyun-Ju Jun, Hye-Ryeong Kang, Hani Koh, Hye Jung Eom, Ji Young Lee, Young Jun Lee, Ji Young Kim, Yun-Young Choi, Kyeongil Park, Mi Jung Kim and Seung-Hyun Kim

    Citation: Journal of Translational Medicine 2017 15:16

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  19. Content type: Research

    Stromal vascular fraction (SVF) can easily be obtained from a mini-lipoaspirate procedure of fat tissue and platelet rich plasma (PRP) can be obtained from peripheral blood. The SVF contains a mixture of cells...

    Authors: Kristin Comella, Robert Silbert and Michelle Parlo

    Citation: Journal of Translational Medicine 2017 15:12

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

  20. Content type: Research

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

    Authors: Dorothea Leonhäuser, Katja Stollenwerk, Volker Seifarth, Isabella M. Zraik, Michael Vogt, Pramod K. Srinivasan, Rene H. Tolba and Joachim O. Grosse

    Citation: Journal of Translational Medicine 2017 15:3

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  21. Content type: Research

    Induced pluripotent stem cells (iPSC) possess an enormous potential as both, scientific and therapeutic tools. Their application in the regenerative medicine provides new treatment opportunities for numerous d...

    Authors: Maciej P. Walczak, Anna M. Drozd, Ewelina Stoczynska-Fidelus, Piotr Rieske and Dawid P. Grzela

    Citation: Journal of Translational Medicine 2016 14:341

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  22. Content type: Research

    Recent studies suggest that immunotherapy using T regulatory cells (Tregs) prolongs remission in type 1 diabetes (T1DM). Here, we report factors that possibly affect the efficacy of this treatment.

    Authors: Natalia Marek-Trzonkowska, Małgorzata Myśliwiec, Dorota Iwaszkiewicz-Grześ, Mateusz Gliwiński, Ilona Derkowska, Magdalena Żalińska, Maciej Zieliński, Marcelina Grabowska, Hanna Zielińska, Karolina Piekarska, Anna Jaźwińska-Curyłło, Radosław Owczuk, Agnieszka Szadkowska, Krystyna Wyka, Piotr Witkowski, Wojciech Młynarski…

    Citation: Journal of Translational Medicine 2016 14:332

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  23. Content type: Research

    Myocardial recovery with left ventricular assist device (LVAD) therapy is highly variable and difficult to predict. Next generation ribonucleic acid (RNA) sequencing is an innovative, rapid, and quantitative a...

    Authors: Kajari Dhar, Alexandra M. Moulton, Eric Rome, Fang Qiu, Jeff Kittrell, Eugenia Raichlin, Ronald Zolty, John Y. Um, Michael J. Moulton, Hesham Basma, Daniel R. Anderson, James D. Eudy and Brian D. Lowes

    Citation: Journal of Translational Medicine 2016 14:327

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  24. Content type: Research

    Adipose derived stromal cells (ASCs) are a rich and convenient source of cells for clinical regenerative therapeutic approaches. However, applications of ASCs often require cell expansion to reach the needed d...

    Authors: Mandana Haack-Sørensen, Bjarke Follin, Morten Juhl, Sonja K. Brorsen, Rebekka H. Søndergaard, Jens Kastrup and Annette Ekblond

    Citation: Journal of Translational Medicine 2016 14:319

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  25. Content type: Research

    Cell-based therapies such as autologous chondrocyte implantation are promising therapeutic approaches to treat cartilage defects to prevent further cartilage degeneration. To assure consistent quality of cell-...

    Authors: Christoph Bartz, Miriam Meixner, Petra Giesemann, Giulietta Roël, Grit-Carsta Bulwin and Jeske J. Smink

    Citation: Journal of Translational Medicine 2016 14:317

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  26. Content type: Research

    The endometrial regenerative cell (ERC) is a novel type of adult mesenchymal stem cell isolated from menstrual blood. Previous studies demonstrated that ERCs possess unique immunoregulatory properties in vitro...

    Authors: Shanzheng Lu, Ganggang Shi, Xiaoxi Xu, Grace Wang, Xu Lan, Peng Sun, Xiang Li, Baoren Zhang, Xiangying Gu, Thomas E. Ichim and Hao Wang

    Citation: Journal of Translational Medicine 2016 14:300

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  27. Content type: Review

    Gene therapy protocols require robust and long-term gene expression. For two decades, retrovirus family vectors have offered several attractive properties as stable gene-delivery vehicles. These vectors repres...

    Authors: José Eduardo Vargas, Leonardo Chicaybam, Renato Tetelbom Stein, Amilcar Tanuri, Andrés Delgado-Cañedo and Martin H. Bonamino

    Citation: Journal of Translational Medicine 2016 14:288

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  28. Content type: Research

    Chronic low back pain due to disc degeneration represents a major social and economic burden worldwide. The current standard of care is limited to symptomatic relief and no current approved therapy promotes di...

    Authors: Christian Elabd, Christopher J. Centeno, John R. Schultz, Gregory Lutz, Thomas Ichim and Francisco J. Silva

    Citation: Journal of Translational Medicine 2016 14:253

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  29. Content type: Research

    Non-healing wounds are a major global health concern and account for the majority of non-traumatic limb amputations worldwide. However, compared to standard care practices, few advanced therapeutics effectivel...

    Authors: Anthony R. Sheets, Conner J. Massey, Stephen M. Cronk, Mark D. Iafrati and Ira M. Herman

    Citation: Journal of Translational Medicine 2016 14:197

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  30. Content type: Research

    Stromal vascular fraction (SVF) can easily be obtained from a mini-lipoaspirate procedure of fat tissue. The SVF contains a mixture of cells including ADSCs and growth factors and has been depleted of the adip...

    Authors: K. Comella, J. Parcero, H. Bansal, J. Perez, J. Lopez, A. Agrawal and T. Ichim

    Citation: Journal of Translational Medicine 2016 14:158

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  31. Content type: Research

    Mesenchymal stromal cells (MSC) are largely investigated in clinical trials aiming to control inappropriate immune reactions (GVHD, Crohn’s disease, solid organ transplantation). As the percentage of MSC precu...

    Authors: Chantal Lechanteur, Alexandra Briquet, Olivier Giet, Olivier Delloye, Etienne Baudoux and Yves Beguin

    Citation: Journal of Translational Medicine 2016 14:145

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  32. Content type: Research

    The trophic, anti-apoptotic and regenerative effects of bone marrow mesenchymal stromal cells (MSC) may reduce neuronal cell loss in neurodegenerative disorders.

    Authors: Margherita Canesi, Rosaria Giordano, Lorenza Lazzari, Maurizio Isalberti, Ioannis Ugo Isaias, Riccardo Benti, Paolo Rampini, Giorgio Marotta, Aurora Colombo, Emanuele Cereda, Mariangela Dipaola, Tiziana Montemurro, Mariele Viganò, Silvia Budelli, Elisa Montelatici, Cristiana Lavazza…

    Citation: Journal of Translational Medicine 2016 14:127

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  33. Content type: Research

    Adipose-derived stem cells (ASCs) have been identified as a population of multipotent cells with promising applications in tissue engineering and regenerative medicine. ASCs are abundant in fat tissue, which c...

    Authors: Dominik Duscher, Anna Luan, Robert C. Rennert, David Atashroo, Zeshaan N. Maan, Elizabeth A. Brett, Alexander J. Whittam, Natalie Ho, Michelle Lin, Michael S. Hu, Graham G. Walmsley, Raphael Wenny, Manfred Schmidt, Arndt F. Schilling, Hans-Günther Machens, Georg M. Huemer…

    Citation: Journal of Translational Medicine 2016 14:126

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  34. Content type: Review

    The mechanisms underlying discrimination between “self” and “non-self”, a central immunological principle, require careful consideration in immune oncology therapeutics where eliciting anti-cancer immunity mus...

    Authors: Samuel C. Wagner, Neil H. Riordan, Thomas E. Ichim, Julia Szymanski, Hong Ma, Jesus A. Perez, Javier Lopez, Juan J. Plata-Munoz, Francisco Silva, Amit N. Patel and Santosh Kesari

    Citation: Journal of Translational Medicine 2016 14:90

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  35. Content type: Research

    Automation of cell therapy manufacturing promises higher productivity of cell factories, more economical use of highly-trained (and costly) manufacturing staff, facilitation of processes requiring manufacturin...

    Authors: Christiane Hümmer, Carolin Poppe, Milica Bunos, Belinda Stock, Eva Wingenfeld, Volker Huppert, Juliane Stuth, Kristina Reck, Mike Essl, Erhard Seifried and Halvard Bonig

    Citation: Journal of Translational Medicine 2016 14:76

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  36. Content type: Research

    High levels of pro-inflammatory cytokines in leukocyte- and platelet-rich plasma (L-PRP) may activate the nuclear factor κB (NF-κB) pathway to counter the beneficial effect of the growth factors on bone regene...

    Authors: Wenjing Yin, Xin Qi, Yuelei Zhang, Jiagen Sheng, Zhengliang Xu, Shicong Tao, Xuetao Xie, Xiaolin Li and Changqing Zhang

    Citation: Journal of Translational Medicine 2016 14:73

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  37. Content type: Research

    Adjuvant imatinib is useful in patients with gastrointestinal stromal tumors (GIST) at high risk of recurrence. At present, the risk of recurrence is determined based on tumor size, mitotic rate, tumor site, a...

    Authors: Keith M. Skubitz, Kate Geschwind, Wayne W. Xu, Joseph S. Koopmeiners and Amy P. N. Skubitz

    Citation: Journal of Translational Medicine 2016 14:51

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  38. Content type: Research

    Orthotopic liver transplantation is the only effective treatment for liver failure but limited with shortage of available donor organs. Recent studies show promising results of mesenchymal stem cells (MSCs)-ba...

    Authors: Biao Huang, Xixi Cheng, Huafeng Wang, Wenjing Huang, Zha la Ga hu, Dan Wang, Kai Zhang, Huan Zhang, Zhenyi Xue, Yurong Da, Ning Zhang, Yongcheng Hu, Zhi Yao, Liang Qiao, Fei Gao and Rongxin Zhang

    Citation: Journal of Translational Medicine 2016 14:45

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  39. Content type: Commentary

    Mesenchymal stromal cells (MSCs) are adult stem cells able to give rise to bone, cartilage and fat cells. In addition, they possess immunomodulatory and immunosuppressive properties that are mainly mediated th...

    Authors: Marie-Theres Zeuner, Ketan Patel, Bernd Denecke, Bernd Giebel and Darius Widera

    Citation: Journal of Translational Medicine 2016 14:34

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  40. Content type: Research

    Endometrial regenerative cells (ERCs) is an attractive novel type of adult mesenchymal stem cells that can be non-invasively obtained from menstrual blood and are easily replicated at a large scale without tum...

    Authors: Peng Sun, Jian Liu, Wenwen Li, Xiaoxi Xu, Xiangying Gu, HongYue Li, Hongqiu Han, Caigan Du and Hao Wang

    Citation: Journal of Translational Medicine 2016 14:28

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  41. Content type: Research

    Craniomaxillofacial defects secondary to trauma, tumor resection, or congenital malformations are frequent unmet challenges, due to suboptimal alloplastic options and limited autologous tissues such as bone. S...

    Authors: Amy L. Strong, Ryan S. Hunter, Robert B. Jones, Annie C. Bowles, Maria F. Dutreil, Dina Gaupp, Daniel J. Hayes, Jeffrey M. Gimble, Benjamin Levi, Margaret A. McNulty and Bruce A. Bunnell

    Citation: Journal of Translational Medicine 2016 14:27

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  42. Content type: Research

    Umbilical cord blood (UCB) is becoming an alternative cell source for hematopoietic stem cell transplantation (HSCT). However, umbilical cord blood transplantation (UCBT) has been severely limited by low and f...

    Authors: Deng-Mei Tian, Ying-Min Liang and Yong-Qing Zhang

    Citation: Journal of Translational Medicine 2016 14:5

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  43. Content type: Research

    Glioblastoma (GBM) is the deadliest of brain tumors. Standard treatment for GBM is surgery, followed by combined radiation therapy and chemotherapy. Current therapy prolongs survival but does not offer a cure....

    Authors: Mark S. Kindy, Jin Yu, Hong Zhu, Michael T. Smith and Sebastiano Gattoni-Celli

    Citation: Journal of Translational Medicine 2016 14:1

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  44. Content type: Review

    Bacterial and fungal infections continue to pose a major clinical challenge in patients with prolonged severe neutropenia after chemotherapy or hematopoietic stem cell transplantation (HSCT). With the advent o...

    Authors: Chiara Cugno, Sara Deola, Perla Filippini, David F. Stroncek and Sergio Rutella

    Citation: Journal of Translational Medicine 2015 13:362

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  45. Content type: Research

    Pooled human platelet lysate (pHPL) is an efficient alternative to xenogenic supplements for ex vivo expansion of mesenchymal stem cells (MSCs) in clinical studies. Currently, porcine heparin is used in pHPL-s...

    Authors: Sandra Laner-Plamberger, Thomas Lener, Doris Schmid, Doris A. Streif, Tina Salzer, Michaela Öller, Cornelia Hauser-Kronberger, Thorsten Fischer, Volker R. Jacobs, Katharina Schallmoser, Mario Gimona and Eva Rohde

    Citation: Journal of Translational Medicine 2015 13:354

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  46. Content type: Protocol

    Mesenchymal stromal cells (MSC) may serve as an attractive therapy in renal transplantation due to their immunosuppressive and reparative properties. While most studies have used autologous MSCs, allogeneic MS...

    Authors: Marlies E. J. Reinders, Geertje J. Dreyer, Jonna R. Bank, Helene Roelofs, Sebastiaan Heidt, Dave L. Roelen, Maarten L. Zandvliet, Volkert A. L. Huurman, Wim E. Fibbe, Cees van Kooten, Frans H. J. Claas, Ton J. Rabelink and Johan W. de Fijter

    Citation: Journal of Translational Medicine 2015 13:344

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  47. Content type: Research

    Demonstrate the safety and effectiveness of highly purified CD133+ autologous stem cells in critical limb ischemia (CLI).

    Authors: Vittorio Arici, Cesare Perotti, Calliada Fabrizio, Claudia Del Fante, Franco Ragni, Francesco Alessandrino, Gianluca Viarengo, Michele Pagani, Alessia Moia, Carmine Tinelli and Antonio Bozzani

    Citation: Journal of Translational Medicine 2015 13:342

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  48. Content type: Review

    Angiogenesis is essential for the growth and metastasis of solid tumors. The tumor endothelium exists in a state of chronic activation and proliferation, fueled by the tumor milieu where angiogenic mediators a...

    Authors: Samuel C. Wagner, Thomas E. Ichim, Hong Ma, Julia Szymanski, Jesus A. Perez, Javier Lopez, Vladimir Bogin, Amit N. Patel, Francisco M. Marincola and Santosh Kesari

    Citation: Journal of Translational Medicine 2015 13:340

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  49. Content type: Research

    Emerging evidence indicates that mesenchymal stromal cells (MSCs) isolated from different tissue sources may be used in vivo as tissue restorative agents. To date, there is no evidence, however, on migration a...

    Authors: Hatixhe Latifi-Pupovci, Zyrafete Kuçi, Sibylle Wehner, Halvard Bönig, Ralf Lieberz, Thomas Klingebiel, Peter Bader and Selim Kuçi

    Citation: Journal of Translational Medicine 2015 13:315

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