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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. CAR T-cell therapy has been recently unveiled as one of the most promising cancer therapies in hematological malignancies. However, solid tumors mount a profound line of defense to escape immunosurveillance by...

    Authors: Khadijeh Alishah, Matthias Birtel, Elham Masoumi, Leila Jafarzadeh, Hamid Reza Mirzaee, Jamshid Hadjati, Ralf-Holger Voss, Mustafa Diken and Sedighe Asad

    Citation: Journal of Translational Medicine 2021 19:482

    Content type: Research

    Published on:

  2. Gene transfer is an important tool for cellular therapies. Lentiviral vectors are most effectively transferred into lymphocytes or hematopoietic progenitor cells using spinoculation. To enable cGMP (current Go...

    Authors: Victoria Ann Remley, Jianjian Jin, Sarmila Sarkar, Larry Moses, Michaela Prochazkova, Yihua Cai, Lipei Shao, Hui Liu, Tatyana Fuksenko, Ping Jin, David F. Stroncek and Steven L. Highfill

    Citation: Journal of Translational Medicine 2021 19:474

    Content type: Research

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  3. Nucleotide mutations in the ABO gene may reduce the activity of glycosyltransferase, resulting in lower levels of A or B antigen expression in red blood cells. Six known splice sites have been identified accordin...

    Authors: Xiaozhen Hong, Yanling Ying, Jingjing Zhang, Shu Chen, Xianguo Xu, Ji He and Faming Zhu

    Citation: Journal of Translational Medicine 2021 19:470

    Content type: Research

    Published on:

  4. Adoptive transfer of chimeric antigen receptor (CAR)-engineered T cells combined with checkpoint inhibition may prevent T cell exhaustion and improve clinical outcomes. However, the approach is limited by cumu...

    Authors: Zhifen Yang, Lingyu Li, Ahu Turkoz, Pohan Chen, Rona Harari-Steinfeld, Maggie Bobbin, Ofir Stefanson, Hana Choi, Violena Pietrobon, Bennett Alphson, Angshumala Goswami, Vitaly Balan, Alper Kearney, Dharmesh Patel, Jin Yang, Damla Inel…

    Citation: Journal of Translational Medicine 2021 19:459

    Content type: Research

    Published on:

  5. HIV-1 reservoirs persist in the presence of combined antiretroviral therapy (cART). However, cART has transformed HIV-1 infection into a chronic disease marked by control of HIV-1 viral load and mortality redu...

    Authors: Matthew Weichseldorfer, Yvonne Affram, Alonso Heredia, Zahra Rikhtegaran-Tehrani, Mohammad M. Sajadi, Sumiko P. Williams, Yutaka Tagaya, Francesca Benedetti, Habib O. Ramadhani, Frank Denaro, Arshi Munawwar, Joseph Bryant, Davide Zella, Marvin Reitz, Fabio Romerio and Olga S. Latinovic

    Citation: Journal of Translational Medicine 2021 19:453

    Content type: Research

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  6. The discovery of the Cystic fibrosis (CF) gene in 1989 has paved the way for incredible progress in treating the disease such that the mean survival age of individuals living with CF is now ~58 years in Canada...

    Authors: Jin-A Lee, Alex Cho, Elena N. Huang, Yiming Xu, Henry Quach, Jim Hu and Amy P. Wong

    Citation: Journal of Translational Medicine 2021 19:452

    Content type: Review

    Published on:

  7. Hypertrophic scar (HTS) is a fibrotic disorder of skins and may have repercussions on the appearance as well as functions of patients. Recent studies related have shown that competitive endogenous RNA (ceRNA) ...

    Authors: Zewei Zhang, Xin Huang, Jiahao Yang, Shuchen Gu, Yixuan Zhao, Yunhan Liu, Yimin Khoong, Shuqi Wang, Shenying Luo, Tao Zan and Guangshuai Li

    Citation: Journal of Translational Medicine 2021 19:451

    Content type: Research

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  8. Established clinical assessments for detecting dementia risk often require time, cost, and face-to-face meetings. We aimed to develop a Simplified Telephone Assessment for Dementia risk (STAD) (a new screening...

    Authors: Keitaro Makino, Sangyoon Lee, Seongryu Bae, Ippei Chiba, Kenji Harada, Osamu Katayama, Yohei Shinkai and Hiroyuki Shimada

    Citation: Journal of Translational Medicine 2021 19:448

    Content type: Research

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  9. In the ovarian follicle, the Theca Cells (TCs) have two main functions: preserving morphological integrity and, importantly, secreting steroid androgen hormones. TCs express the essential enzyme 17α-hydroxylas...

    Authors: Christian Secchi, Martina Belli, Tracy N. H. Harrison, Joseph Swift, CheMyong Ko, Antoni J. Duleba, Dwayne Stupack, R. Jeffrey Chang and Shunichi Shimasaki

    Citation: Journal of Translational Medicine 2021 19:428

    Content type: Research

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  10. The present study aimed to compare the efficacy and safety profiles of microspheres versus (vs.) polyvinyl alcohol (PVA) for bronchial artery embolization (BAE) treatment in patients with hemoptysis.

    Authors: Zhigang Fu, Xun Li, Fei Cai, Yinpeng Yuan, Xiaolin Zhang, Jingxia Qin and Yonghui Liang

    Citation: Journal of Translational Medicine 2021 19:422

    Content type: Research

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  11. 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

    Content type: Research

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  12. 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

    Content type: Review

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  13. 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

    Content type: Methodology

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  14. 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

    Content type: Commentary

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  15. 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

    Content type: Research

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  16. 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

    Content type: Review

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  17. 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

    Content type: Research

    Published on:

  18. 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

    Content type: Review

    Published on:

  19. 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

    Content type: Research

    Published on:

  20. 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

    Content type: Research

    Published on:

  21. 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

    Content type: Review

    Published on:

  22. 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

    Content type: Review

    Published on:

  23. 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

    Content type: Review

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  24. 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

    Content type: Research

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  25. 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

    Content type: Research

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  26. 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

    Content type: Research

    Published on:

  27. 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:

  28. 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

    Published on:

  29. 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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  30. 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

    Published on:

  31. 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

    Published on:

  32. 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

    Published on:

  33. 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

    Published on:

  34. 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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  35. 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

    Published on:

  36. 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

    Published on:

  37. 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:

  38. 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:

  39. 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

    Published on:

  40. 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:

  41. 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

    Published on:

  42. 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

    Published on:

  43. 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

    Published on:

  44. 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

    Published on:

  45. 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

    Published on:

  46. 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

    Published on:

  47. 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

    Published on:

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