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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. Although chimeric antigen receptor (CAR)-T cell therapy has been remarkably successful for haematological malignancies, its efficacy against solid tumors is limited. The combination of CAR-T cell therapy with ...

    Authors: Yajun Zhang, Pei Wang, Tengjiao Wang, Yuan Fang, Yongmei Ding and Qijun Qian

    Citation: Journal of Translational Medicine 2021 19:82

    Content type: Research

    Published on:

  2. Skeletal muscle aging is associated with a decline in motor function and loss of muscle mass- a condition known as sarcopenia. The underlying mechanisms that drive this pathology are associated with a failure ...

    Authors: Colin Harper, Venkatesh Gopalan and Jorming Goh

    Citation: Journal of Translational Medicine 2021 19:71

    Content type: Review

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  3. Cutaneous wounds in patients with diabetes exhibit impaired healing due to physiological impediments and conventional care options are severely limited. Multipotent stromal cells (MSCs) have been touted as a p...

    Authors: Bibi S. Subhan, Jennifer Kwong, Joseph F. Kuhn, Arie Monas, Sonali Sharma and Piul S. Rabbani

    Citation: Journal of Translational Medicine 2021 19:16

    Content type: Research

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  4. Currently, there are limited reports regarding investigation of the biological properties of polyetheretherketone (PEEK) coated with titanium (Ti) and hydroxyapatite (HA) in human. The objective of this study ...

    Authors: Ce Zhu, Miaomiao He, Lili Mao, Tao Li, Li Zhang, Limin Liu, Ganjun Feng and Yueming Song

    Citation: Journal of Translational Medicine 2021 19:14

    Content type: Research

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  5. Healthy volunteer registry donors have become the backbone of stem cell transplantation programs. While most registrants will never become actual donors, a small minority are called upon twice, most commonly f...

    Authors: Soo-Zin Kim-Wanner, Seo-Youn Lee, Erhard Seifried and Halvard Bonig

    Citation: Journal of Translational Medicine 2020 18:487

    Content type: Research

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  6. Myeloid-derived suppressor cells (MDSCs) play a critical role in modulating the immune response and promoting immune tolerance in models of autoimmunity and transplantation. Regulatory T cells (Tregs) exert th...

    Authors: Min-Jung Park, Jin-Ah Baek, Se-Young Kim, Kyung-Ah Jung, Jeong Won Choi, Sung-Hwan Park, Seung‐Ki Kwok and Mi-La Cho

    Citation: Journal of Translational Medicine 2020 18:483

    Content type: Research

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  7. Periodontitis, a bacterium-induced inflammatory disease that is characterized by alveolar bone loss, is highly prevalent worldwide. Elucidating the underlying mechanisms of alveolar bone loss in periodontitis ...

    Authors: Xiaofei Huang, Mengru Xie, Yanling Xie, Feng Mei, Xiaofeng Lu, Xiaoshuang Li and Lili Chen

    Citation: Journal of Translational Medicine 2020 18:479

    Content type: Review

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  8. During the coronavirus disease-2019 (COVID-19) pandemic, Italian hospitals faced the most daunting challenges of their recent history, and only essential therapeutic interventions were feasible. From March to ...

    Authors: Giuseppe Astori, Martina Bernardi, Angela Bozza, Daniela Catanzaro, Katia Chieregato, Anna Merlo, Monica Santimaria, Roberto Barbazza, Giuseppe Amodeo, Rachele Ciccocioppo, Francesca Elice and Marco Ruggeri

    Citation: Journal of Translational Medicine 2020 18:451

    Content type: Research

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  9. Mesenchymal stem cells (MSCs) have captured great attention in regenerative medicine for over a few decades by virtue of their differentiation capacity, potent immunomodulatory properties, and their ability to...

    Authors: Sepideh Nikfarjam, Jafar Rezaie, Naime Majidi Zolbanin and Reza Jafari

    Citation: Journal of Translational Medicine 2020 18:449

    Content type: Review

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  10. Vectors derived from adeno-associated viruses (AAVs) are widely used for gene transfer both in vitro and in vivo and have gained increasing interest as shuttle systems to deliver therapeutic genes to the heart...

    Authors: Zihou Liu, Kristin Klose, Sebastian Neuber, Meng Jiang, Manfred Gossen and Christof Stamm

    Citation: Journal of Translational Medicine 2020 18:437

    Content type: Methodology

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  11. Extracellular vesicles are heterogeneous populations of naturally occurring secreted small vesicles. EVs function as signaling platforms to facilitate intracellular communication, which indicates the physiolog...

    Authors: Kaiyuan Xu, Qin Liu, Kaihui Wu, Liu Liu, Maomao Zhao, Hui Yang, Xiang Wang and Wenmei Wang

    Citation: Journal of Translational Medicine 2020 18:432

    Content type: Review

    Published on:

  12. Glioblastoma multiforme (GBM) is the most common malignant brain cancer that invades normal brain tissue and impedes surgical eradication, resulting in early local recurrence and high mortality. In addition, m...

    Authors: Craig A. Land, Phillip R. Musich, Dalia Haydar, Giedre Krenciute and Qian Xie

    Citation: Journal of Translational Medicine 2020 18:428

    Content type: Review

    Published on:

  13. Cancer vaccines provide a complex source of neoantigens. Still, increasing evidence reveals that the neoantigen quality rather than the quantity is predictive for treatment outcome.

    Authors: Inken Salewski, Yvonne Saara Gladbach, Steffen Kuntoff, Nina Irmscher, Olga Hahn, Christian Junghanss and Claudia Maletzki

    Citation: Journal of Translational Medicine 2020 18:402

    Content type: Research

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  14. Osteonecrosis (ON) is an acquired debilitating skeletal disorder, which is caused by a multitude of traumatic and non-traumatic etiological factors. Vascular damage, mechanical stress and increased intraosseou...

    Authors: S. Elgaz, H. Bonig and P. Bader

    Citation: Journal of Translational Medicine 2020 18:399

    Content type: Review

    Published on:

  15. Bronchopulmonary dysplasia (BPD) is a severe condition in premature infants that compromises lung function and necessitates oxygen support. Despite major improvements in perinatal care minimizing the devastati...

    Authors: Liem Thanh Nguyen, Thai T. H. Trieu, Hue T. H. Bui, Van T. Hoang, Anh T. T. Nguyen, Nhung T. H. Trinh, Kien T. Nguyen and Duc M. Hoang

    Citation: Journal of Translational Medicine 2020 18:398

    Content type: Research

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  16. Very preterm infants are at risk of developing retinopathy of prematurity (ROP). Recombinant human erythropoietin (rhEPO) is routinely used to prevent anemia in preterm infants; however, the effect of rhEPO on...

    Authors: Huiqing Sun, Juan Song, Wenqing Kang, Yong Wang, Xiantao Sun, Chongchen Zhou, Hong Xiong, Falin Xu, Mingchao Li, Xiaoli Zhang, Zengyuan Yu, Xirui Peng, Bingbing Li, Yiran Xu, Shan Xing, Xiaoyang Wang…

    Citation: Journal of Translational Medicine 2020 18:397

    Content type: Research

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  17. A major obstacle to anti-viral and -tumor cell vaccination and T cell immunotherapy is the ability to produce dendritic cells (DCs) in a suitable clinical setting. It is imperative to develop closed cell cultu...

    Authors: Jean-Philippe Bastien, Natalie Fekete, Ariane V. Beland, Marie-Paule Lachambre, Veronique Laforte, David Juncker, Vibhuti Dave, Denis-Claude Roy and Corinne A. Hoesli

    Citation: Journal of Translational Medicine 2020 18:383

    Content type: Research

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  18. The existence of active crosstalk between cells in a paracrine and juxtacrine manner dictates specific activity under physiological and pathological conditions. Upon juxtacrine interaction between the cells, v...

    Authors: Hesam Saghaei Bagheri, Farhad Bani, Savas Tasoglu, Amir Zarebkohan, Reza Rahbarghazi and Emel Sokullu

    Citation: Journal of Translational Medicine 2020 18:367

    Content type: Review

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  19. Adoptive transfer of engineered immune cells is a promising strategy for cancer treatment. However, low transduction efficiency particularly when large payload lentiviral vectors are used on primary T cells is...

    Authors: Lingyu Li, Yuan Gao, Richa Srivastava, Wei Wang, Qinghui Xiong, Zhiming Fang, Alejandra Pelayo, Carolyn Denson, Angshumala Goswami, Rona Harari-Steinfeld, Zhifen Yang, Lihong Weng, Lei Stanley Qi and Francesco M. Marincola

    Citation: Journal of Translational Medicine 2020 18:363

    Content type: Research

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  20. Extracellular vesicles (ECV) and bone extracellular matrix (ECM) have beneficial effects on the treatment of some pathological conditions. The purpose of this study was to find the synergic effects of decellul...

    Authors: Asrin Emami, Tahereh Talaei-Khozani, Saeid Tavanafar, Nehleh Zareifard, Negar Azarpira and Zahra Vojdani

    Citation: Journal of Translational Medicine 2020 18:361

    Content type: Research

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  21. Mesenchymal stromal cells are a safe and promising option to treat knee osteoarthritis as previously demonstrated in different clinical trials. However, their efficacy, optimal dose and addition of adjuvants m...

    Authors: José María Lamo-Espinosa, Juan F. Blanco, Mikel Sánchez, Victoria Moreno, Froilán Granero-Moltó, Fermín Sánchez-Guijo, Íñigo Crespo-Cullel, Gonzalo Mora, Diego Delgado San Vicente, Orlando Pompei-Fernández, Jesús Dámaso Aquerreta, Jorge María Núñez-Córdoba, María Vitoria Sola, Andrés Valentí-Azcárate, Enrique J. Andreu, María del Consuelo del Cañizo…

    Citation: Journal of Translational Medicine 2020 18:356

    Content type: Research

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  22. Foetal bovine serum (FBS), is the most commonly used culture medium additive for in vitro cultures, despite its undefined composition, its potential immunogenicity and possible prion/zoonotic transmission. For...

    Authors: M. Guiotto, W. Raffoul, A. M. Hart, M. O. Riehle and P. G. di Summa

    Citation: Journal of Translational Medicine 2020 18:351

    Content type: Review

    Published on:

  23. Deceased organ donors represent an untapped source of therapeutic bone marrow (BM) that can be recovered in 3–5 times the volume of that obtained from living donors, tested for quality, cryopreserved, and bank...

    Authors: Erik J. Woods, Aubrey M. Sherry, John R. Woods, James W. Hardin, Michael LaFontaine, Gerald Brandacher and Brian H. Johnstone

    Citation: Journal of Translational Medicine 2020 18:300

    Content type: Research

    Published on:

  24. Treatment of hepatocellular carcinoma (HCC) using antibody-based targeted therapies, such as antibody conjugates and chimeric antigen receptor T (CAR-T) cell therapy, shows potent antitumor efficacy. Glypican-...

    Authors: Xiaoyu Liu, Fang Gao, Longwei Jiang, Meng Jia, Lei Ao, Ming Lu, Liming Gou, Mitchell Ho, Shaochang Jia, Fei Chen and Wei Gao

    Citation: Journal of Translational Medicine 2020 18:295

    Content type: Research

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  25. Although a large body of information exists relating to cellular therapies, much of this information is either anecdotal or has been obtained from relatively small clinical trials, so that the level of evidenc...

    Authors: Amanda Lindeman, Carl J. Pepine and Keith L. March

    Citation: Journal of Translational Medicine 2020 18:285

    Content type: Research

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  26. Mesenchymal stem cells (MSCs) have been shown to alleviate acute lung injury (ALI) and induce the production of regulatory dendritic cells (DCregs), but the potential link between these two cell types remains ...

    Authors: Zhonghua Lu, Shanshan Meng, Wei Chang, Shanwen Fan, Jianfeng Xie, Fengmei Guo, Yi Yang, Haibo Qiu and Ling Liu

    Citation: Journal of Translational Medicine 2020 18:241

    Content type: Research

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  27. The in vitro production of mature human red blood cells (RBCs) from induced pluripotent stem cells (iPSCs) has been the focus of research to meet the high demand for blood transfusions. However, limitations li...

    Authors: Yu Jin Park, Su-Hee Jeon, Hyun-Kyung Kim, Eun Jung Suh, Seung Jun Choi, Sinyoung Kim and Hyun Ok Kim

    Citation: Journal of Translational Medicine 2020 18:236

    Content type: Research

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  28. Typically minor ABO incompatible platelet products are transfused without any incident, yet serious hemolytic transfusion reactions occur. To mitigate these events, ABO ‘low titer’ products are used for minor ...

    Authors: Priyanka Pandey, Waseem Q. Anani, Tina Pugh, Jerome L. Gottschall and Gregory A. Denomme

    Citation: Journal of Translational Medicine 2020 18:216

    Content type: Research

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  29. In late 2019, a novel coronavirus (SARS-CoV-2) emerged in Wuhan, capital city of Hubei province in China. Cases of SARS-CoV-2 infection quickly grew by several thousand per day. Less than 100 days later, the W...

    Authors: Christopher J. Rogers, Robert J. Harman, Bruce A. Bunnell, Martin A. Schreiber, Charlie Xiang, Fu-Sheng Wang, Antonio F. Santidrian and Boris R. Minev

    Citation: Journal of Translational Medicine 2020 18:203

    Content type: Review

    Published on:

  30. Glioblastoma stem-like cells (GSCs) are hypothesized to contribute to self-renewal and therapeutic resistance in glioblastoma multiforme (GBM) tumors. Constituting only a small percentage of cancer cells, GSCs...

    Authors: Xu Wang, Wenjuan Zhou, Xian Li, Jun Ren, Guangyu Ji, Jingyi Du, Wenyu Tian, Qian Liu and Aijun Hao

    Citation: Journal of Translational Medicine 2020 18:200

    Content type: Research

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  31. We present the rationale for a novel allo-priming approach to serve the elderly as a universal anti-virus vaccine, as well serving to remodel the aging immune system in order to reverse immunosenescence and in...

    Authors: Michael Har-Noy and Reuven Or

    Citation: Journal of Translational Medicine 2020 18:196

    Content type: Review

    Published on:

  32. Genetically engineered T cells have become an important therapy for B-cell malignancies. Measuring the efficiency of vector integration into the T cell genome is important for assessing the potency and safety ...

    Authors: Alex Lu, Hui Liu, Rongye Shi, Yihua Cai, Jinxia Ma, Lipei Shao, Victor Rong, Nikolaos Gkitsas, Hong Lei, Steven L. Highfill, Sandhya Panch, David F. Stroncek and Ping Jin

    Citation: Journal of Translational Medicine 2020 18:191

    Content type: Research

    Published on:

  33. Progressive multifocal leukoencephalopathy is a demyelinating CNS disorder. Reactivation of John Cunningham virus leads to oligodendrocyte infection with lysis and consequent axonal loss due to demyelination. ...

    Authors: M. J. Steinhardt, E. Wiercinska, M. Pham, G. U. Grigoleit, A. Mazzoni, M. Da-Via, X. Zhou, K. Meckel, K. Nickel, J. Duell, F. C. Krummenast, S. Kraus, C. Hopkinson, B. Weissbrich, W. Müllges, G. Stoll…

    Citation: Journal of Translational Medicine 2020 18:177

    Content type: Methodology

    Published on:

  34. Kidney ischemia reperfusion injury (IRI) is a common cause of acute kidney injury and an unavoidable consequence of kidney transplantation and still lacks specific therapeutics. Recently, mesenchymal stem cell...

    Authors: Shuo Wang, Songjie Cai, Weitao Zhang, Xigao Liu, Yan Li, Chao Zhang, Yigang Zeng, Ming Xu, Ruiming Rong, Tianshu Yang, Benkang Shi, Anil Chandraker, Cheng Yang and Tongyu Zhu

    Citation: Journal of Translational Medicine 2020 18:175

    Content type: Research

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  35. Adoptive transfer of virus-specific T cells (VSTs) represents a prophylactic and curative approach for opportunistic viral infections and reactivations after transplantation. However, inadequate frequencies of...

    Authors: Nele Carolin Heinemann, Sabine Tischer-Zimmermann, Torge Christian Wittke, Julian Eigendorf, Arno Kerling, Theodor Framke, Anette Melk, Hans-Gert Heuft, Rainer Blasczyk, Britta Maecker-Kolhoff and Britta Eiz-Vesper

    Citation: Journal of Translational Medicine 2020 18:148

    Content type: Research

    Published on:

  36. The skin is the largest organ of the human body. Upon injury, the skin triggers a sequence of signaling pathways that induce epithelial proliferation, migration, and ultimately, the re-establishment of the epi...

    Authors: Carlos H. V. Nascimento-Filho, Ericka J. D. Silveira, Eny M. Goloni-Bertollo, Lelia Batista de Souza, Cristiane H. Squarize and Rogerio M. Castilho

    Citation: Journal of Translational Medicine 2020 18:138

    Content type: Research

    Published on:

  37. Cell proliferation and death are key components of wound healing and tissue repair. Telocytes (TCs) represent a newly discovered cell type that can protect tissue from acute injury via cell–cell communication ...

    Authors: Lu Wang, Dongli Song, Chuanyuan Wei, Cheng Chen, Yanwen Yang, Xinyi Deng and Jianying Gu

    Citation: Journal of Translational Medicine 2020 18:60

    Content type: Research

    Published on:

  38. Tissue-engineered skin (TES), as an analogue of native skin, is promising for wound repair and regeneration. However, a major drawback of TES products is a lack of skin appendages and nerves to enhance skin he...

    Authors: Tingting Weng, Pan Wu, Wei Zhang, Yurong Zheng, Qiong Li, Ronghua Jin, Haojiao Chen, Chuangang You, Songxue Guo, Chunmao Han and Xingang Wang

    Citation: Journal of Translational Medicine 2020 18:53

    Content type: Review

    Published on:

  39. Mesenchymal stromal cells (MSCs) have attracted intense interest due to their powerful intrinsic properties of self-regeneration, immunomodulation and multi-potency, as well as being readily available and easy...

    Authors: Dickson Kofi Wiredu Ocansey, Bing Pei, Yongmin Yan, Hui Qian, Xu Zhang, Wenrong Xu and Fei Mao

    Citation: Journal of Translational Medicine 2020 18:42

    Content type: Review

    Published on:

  40. Bone marrow mononuclear cells have been successfully utilized for numerous regenerative purposes. In the current study, patients suffering from erectile dysfunction (ED) unresponsive to phosphodiesterase 5 inh...

    Authors: Mark Bieri, Elias Said, Gabrielle Antonini, Donald Dickerson, Jorge Tuma, Courtney E. Bartlett, Amit N. Patel and Alexander Gershman

    Citation: Journal of Translational Medicine 2020 18:24

    Content type: Research

    Published on:

  41. Prostate cancer is one of the most common adult malignancies in men, and nearly all patients with metastatic prostate cancer can develop and receive resistance to primary androgen deprivation therapy (ADT), a ...

    Authors: Dawei Wang, Yuan Shao, Xiang Zhang, Guoliang Lu and Boke Liu

    Citation: Journal of Translational Medicine 2020 18:23

    Content type: Research

    Published on:

  42. Malignant behavior and radioresistance, which severely limits the efficacy of radiation therapy (RT) in nasopharyngeal carcinoma (NPC), are associated with tumor progression and poor prognosis. Mesenchymal ste...

    Authors: Fang-Zhu Wan, Kai-Hua Chen, Yong-Chu Sun, Xi-Chan Chen, Ren-Ba Liang, Li Chen and Xiao-Dong Zhu

    Citation: Journal of Translational Medicine 2020 18:12

    Content type: Research

    Published on:

  43. A worldwide lack of donor corneas demands the bioengineered corneas be developed as an alternative. The primary objective of the current study was to evaluate the efficacy of acellular porcine corneal stroma (...

    Authors: Saiqun Li, Meng Li, Li Gu, Lulu Peng, Yuqing Deng, Jing Zhong, Bowen Wang, Qian Wang, Yichen Xiao and Jin Yuan

    Citation: Journal of Translational Medicine 2019 17:434

    Content type: Research

    Published on:

  44. Innovative human stromal cell therapeutics require xeno-free culture conditions. Various formulations of human platelet lysate (HPL) are efficient alternatives for fetal bovine serum (FBS). However, a consiste...

    Authors: Sandra Laner-Plamberger, Michaela Oeller, Cornelia Mrazek, Arnulf Hartl, Alina Sonderegger, Eva Rohde, Dirk Strunk and Katharina Schallmoser

    Citation: Journal of Translational Medicine 2019 17:432

    Content type: Research

    Published on:

  45. Telocytes (TCs) have the capacity of cell–cell communication with adjacent cells within the tissue, contributing to tissue repair and recovery from injury. The present study aims at investigating the molecular...

    Authors: Dongli Song, Li Tang, Jianan Huang, Lu Wang, Tao Zeng and Xiangdong Wang

    Citation: Journal of Translational Medicine 2019 17:431

    Content type: Research

    Published on:

  46. Delayed bone healing, especially in long bones poses one of the biggest problems in orthopeadic and reconstructive surgery and causes tremendous costs every year. There is a need for exploring the causes in or...

    Authors: Johannes M. Wagner, Sonja V. Schmidt, Mehran Dadras, Julika Huber, Christoph Wallner, Stephanie Dittfeld, Mustafa Becerikli, Henriette Jaurich, Felix Reinkemeier, Marius Drysch, Marcus Lehnhardt and Björn Behr

    Citation: Journal of Translational Medicine 2019 17:416

    Content type: Research

    Published on:

  47. The liver is supplied by a dual blood supply, including the portal venous system and the hepatic arterial system; thus, the liver organ is exposed to multiple gut microbial products, metabolic products, and to...

    Authors: Chenxia Hu and Lanjuan Li

    Citation: Journal of Translational Medicine 2019 17:412

    Content type: Review

    Published on:

  48. Mesenchymal stem cells (MSCs) represent an invaluable asset for the field of cell therapy. Human Bone marrow-derived MSCs (hBM-MSCs) are one of the most commonly used cell types in clinical trials. They are cu...

    Authors: Soukaina Bahsoun, Karen Coopman and Elizabeth C. Akam

    Citation: Journal of Translational Medicine 2019 17:397

    Content type: Review

    Published on:

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