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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. Peripheral blood mononuclear cells (PBMC) concentrates collected by apheresis are frequently used as starting material for cellular therapies, but the cell of interest must often be isolated prior to initiatin...

    Authors: David F Stroncek, Vicki Fellowes, Chauha Pham, Hanh Khuu, Daniel H Fowler, Lauren V Wood and Marianna Sabatino
    Citation: Journal of Translational Medicine 2014 12:241
    Content type: Research Published on:

  2. At present, no effective clinical treatment is available for the late effects of radiation myelopathy. The aim of the present study was to assess the therapeutic effects of human umbilical cord-derived mesench...

    Authors: Li Wei, Jing Zhang, Xiu-Bin Xiao, Hai-Xing Mai, Ke Zheng, Wan-Liang Sun, Lei Wang, Feng Liang, Zai-Liang Yang, Yuan Liu, Yan-Qing Wang, Zhi-Fang Li, Jia-Ning Wang, Wei-Jing Zhang and Hua You
    Citation: Journal of Translational Medicine 2014 12:246
    Content type: Research Published on:

  3. Aberrant expression of miR-1 has been implicated in various cancers. However, the mechanisms underlying the role of miR-1 in CRC progression still have not been clarified clearly. Here, we showed the decreased...

    Authors: Lijun Xu, Yue Zhang, Hui Wang, Guanhua Zhang, Yanqing Ding and Liang Zhao
    Citation: Journal of Translational Medicine 2014 12:244
    Content type: Research Published on:

  4. The inadequacy of existing therapeutic tools together with the paucity of organ donors have always led medical researchers to innovate the current treatment methods or to discover new ways to cure disease. Eme...

    Authors: Thekkeparambil Chandrabose Srijaya, Thamil Selvee Ramasamy and Noor Hayaty Abu Kasim
    Citation: Journal of Translational Medicine 2014 12:243
    Content type: Review Published on:

  5. 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
    Content type: Research Published on:

  6. 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
    Content type: Review Published on:

  7. 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
    Content type: Research Published on:

  8. 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
    Content type: Research Published on:

  9. 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
    Content type: Research Published on:

  10. 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
    Content type: Research Published on:

  11. 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
    Content type: Research Published on:

  12. 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
    Content type: Research Published on:

  13. 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
    Content type: Research Published on:

  14. 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
    Content type: Research Published on:

  15. 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
    Content type: Research Published on:

  16. 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
    Content type: Review Published on:

  17. 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
    Content type: Research Published on:

  18. 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
    Content type: Research Published on:

  19. 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
    Content type: Research Published on:

  21. 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
    Content type: Research Published on:

  23. 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
    Content type: Research Published on:

  24. 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
    Content type: Research Published on:

  25. 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
    Content type: Research Published on:

  26. 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
    Content type: Research Published on:

  27. 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
    Content type: Research Published on:

  28. 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
    Content type: Research Published on:

  29. 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
    Content type: Research Published on:

  30. 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
    Content type: Research Published on:

  31. 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
    Content type: Research Published on:

  32. 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
    Content type: Research Published on:

  33. 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
    Content type: Research Published on:

  34. 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
    Content type: Research Published on:

  35. 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
    Content type: Review Published on:

  36. 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
    Content type: Research Published on:

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

  37. 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
    Content type: Research Published on:

  38. 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
    Content type: Research Published on:

  39. 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
    Content type: Research Published on:

  40. 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
    Content type: Research Published on:

  41. 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
    Content type: Research Published on:

  42. 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
    Content type: Research Published on:

  43. 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
    Content type: Research Published on:

  44. 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
    Content type: Research Published on:

  45. 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
    Content type: Research Published on:

  46. 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
    Content type: Research Published on:

  47. 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
    Content type: Research Published on:

  48. 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
    Content type: Methodology Published on:

  49. 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
    Content type: Research Published on:

  50. 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
    Content type: Research Published on:
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