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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. Systemic sclerosis is an autoimmune disease characterized by excessive dermal fibrosis with progression to internal organs, vascular impairment and immune dysregulation evidenced by the infiltration of inflamm...

    Authors: Pauline Beurier, Laure Ricard, Deborah Eshagh, Florent Malard, Lama Siblany, Olivier Fain, Mohamad Mohty, Béatrice Gaugler and Arsène Mekinian
    Citation: Journal of Translational Medicine 2021 19:375
  2. Recently, mesenchymal stem/stromal cells (MSCs) due to their pro-angiogenic, anti-apoptotic, and immunoregulatory competencies along with fewer ethical issues are presented as a rational strategy for regenerat...

    Authors: Soudeh Moghadasi, Marischa Elveny, Heshu Sulaiman Rahman, Wanich Suksatan, Abduladheem Turki Jalil, Walid Kamal Abdelbasset, Alexei Valerievich Yumashev, Siavash Shariatzadeh, Roza Motavalli, Farahnaz Behzad, Faroogh Marofi, Ali Hassanzadeh, Yashwant Pathak and Mostafa Jarahian
    Citation: Journal of Translational Medicine 2021 19:302
  3. Genetically engineered chimeric antigen receptor (CAR) T lymphocytes are promising therapeutic tools for cancer. Four CAR T cell drugs, including tisagenlecleucel (tisa-cel) and axicabtagene-ciloleucel (axi-ce...

    Authors: Rafik Haderbache, Walid Warda, Eric Hervouet, Mathieu Neto da Rocha, Rim Trad, Vincent Allain, Clementine Nicod, Catherine Thieblemeont, Nicolas Boissel, Pauline Varlet, Ibrahim Yakoub Agha, Lucie Bouquet, Melanie Guiot, Fabienne Venet, Pierre Sujobert, Xavier Roussel…
    Citation: Journal of Translational Medicine 2021 19:265
  4. Adipose cell-free derivatives have been recently gaining attention as potential therapeutic agents for various human diseases. In this context, mesenchymal stromal/stem cells (MSCs), adipocyte mesenchymal stem...

    Authors: Loubna Mazini, Luc Rochette and Gabriel Malka
    Citation: Journal of Translational Medicine 2021 19:234
  5. Transfusion-related immunomodulation (TRIM) can be caused by exosomes, in which case, microRNAs (miRNAs) are one critical factor impacting exosome behavior. This study aims to investigate and analyze the expre...

    Authors: Yujie Kong, Xue Tian, Rui He, Chenyue Li, Haixia Xu, Li Tian and Zhong Liu
    Citation: Journal of Translational Medicine 2021 19:225
  6. Mitochondria are essential organelles that provide energy for cellular functions, participate in cellular signaling and growth, and facilitate cell death. Based on their multifactorial roles, mitochondria are ...

    Authors: Kei Hayashida, Ryosuke Takegawa, Muhammad Shoaib, Tomoaki Aoki, Rishabh C. Choudhary, Cyrus E. Kuschner, Mitsuaki Nishikimi, Santiago J. Miyara, Daniel M. Rolston, Sara Guevara, Junhwan Kim, Koichiro Shinozaki, Ernesto P. Molmenti and Lance B. Becker
    Citation: Journal of Translational Medicine 2021 19:214
  7. Due to mTOR (mammalian/mechanistic target of rapamycin) gene-loss mice die during embryonic development, the role of mTOR in platelets has not been evaluated using gene knockout technology.

    Authors: Longsheng Wang, Gang Liu, Nannan Wu, Baiyun Dai, Shuang Han, Qiaoyun Liu, Fang Huang, Zhihua Chen, Weihong Xu, Dajing Xia and Cunji Gao
    Citation: Journal of Translational Medicine 2021 19:201
  8. The mortality rate of critically ill patients with acute respiratory distress syndrome (ARDS) is 30.9% to 46.1%. The emergence of the coronavirus disease 2019 (Covid-19) has become a global issue with raising ...

    Authors: Wendi Wang, Wei Lei, Lina Jiang, Siqi Gao, Shijun Hu, Zi-Gang Zhao, Chun-Yu Niu and Zhen-Ao Zhao
    Citation: Journal of Translational Medicine 2021 19:198
  9. Endogenous Peptide Inhibitor of CXCR4 (EPI-X4) is a natural antagonist of the CXC chemokine receptor 4 (CXCR4). EPI-X4 is a 16-mer peptide that is released from human serum albumin (HSA) by acidic aspartic pro...

    Authors: Andrea Gilg, Mirja Harms, Lia-Raluca Olari, Ann-Kathrin Urbanowitz, Halvard Bonig and Jan Münch
    Citation: Journal of Translational Medicine 2021 19:190
  10. Cetuximab has been approved for use for first-line treatment of patients with wild-type KRAS metastatic colorectal cancer (CRC). However, treatment with cetuximab has shown limited efficacy as a CRC monotherapy. ...

    Authors: Shiqi Long, Yangzhuo Gu, Yuanyuan An, Xiaojin Lin, Xiaoqing Chen, Xianyao Wang, Chunxiang Liao, Weiwei Ouyang, Nianxue Wang, Zhixu He and Xing Zhao
    Citation: Journal of Translational Medicine 2021 19:185
  11. Spinal cord injury (SCI) is a severe traumatic disease of the central nervous system, with a global prevalence of 236–4187 per million people. This meta-analysis aimed to evaluate the safety and efficacy of me...

    Authors: Wei-can Chen, Wei-feng Liu, Yu-yan Bai, Ying-ying Zhou, Yan Zhang, Cong-mei Wang, Shu Lin and He-fan He
    Citation: Journal of Translational Medicine 2021 19:178
  12. The onset of the SARS-CoV-2 pandemic has resulted in ever-increasing casualties worldwide, and after 15 months, standard therapeutic regimens are yet to be discovered.

    Authors: Monire Jamalkhah, Yasaman Asaadi, Mohammadreza Azangou-Khyavy, Javad Khanali, Masoud Soleimani, Jafar Kiani and Ehsan Arefian
    Citation: Journal of Translational Medicine 2021 19:164
  13. Multipotent mesenchymal stromal cells (MSCs) are promising cellular therapeutics for the treatment of inflammatory and degenerative disorders due to their anti-inflammatory, immunomodulatory and regenerative p...

    Authors: Nerea Cuesta-Gomez, Gerard J. Graham and John D. M. Campbell
    Citation: Journal of Translational Medicine 2021 19:156
  14. Mesenchymal stem cells derived from human umbilical cord (hUC-MSCs) have immunomodulatory properties that are of interest to treat novel coronavirus disease 2019 (COVID-19). Leng et al. recently reported that ...

    Authors: Jonathan J. Hernandez, Doyle E. Beaty, Logan L. Fruhwirth, Ana P. Lopes Chaves and Neil H. Riordan
    Citation: Journal of Translational Medicine 2021 19:149
  15. Because of limitations of transportation imposed by the COVID-19 pandemic, current recommendation calls for cryopreservation of allogeneic stem cell transplants before patient conditioning. A single cell thera...

    Authors: Eliza Wiercinska, Vera Schlipfenbacher, Gesine Bug, Peter Bader, Mareike Verbeek, Erhard Seifried and Halvard Bonig
    Citation: Journal of Translational Medicine 2021 19:145
  16. Glioma is a complex cancer with a high morbidity and high mortality. Bone marrow mesenchymal stem cells (BMSCs) have shown promise as an excellent cell/drug delivery vehicle for gene-targeted therapy; however,...

    Authors: Jianjun Wu, Shoupin Xie, Hailong Li, Yanxia Zhang, Jia Yue, Chunlu Yan, Kai Liu, Yongqi Liu, Rui Xu and Guisen Zheng
    Citation: Journal of Translational Medicine 2021 19:143
  17. 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
  18. 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
  19. 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
  20. 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
  21. 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
  22. 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
  23. 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
  24. 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
  25. 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
  26. 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
  27. 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
  28. 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
  29. 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
  30. 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
  31. 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
  32. 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
  33. 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
  34. 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
  35. 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
  36. 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
  37. 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
  38. 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
  39. 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
  40. 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
  41. 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
  42. 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
  43. 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
  44. 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
  45. 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
  46. 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
  47. 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

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